BMRN BioMarin Pharmaceutical Inc.

77.07
-1.16  -1%
Previous Close 78.23
Open 78.02
52 Week Low 71.35
52 Week High 131.945
Market Cap $14,013,613,438
Shares 181,829,680
Float 159,007,864
Enterprise Value $14,246,048,866
Volume 767,016
Av. Daily Volume 1,200,911
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Upcoming Catalysts

Drug Stage Catalyst Date
Vosoritide
Achondroplasia
PDUFA
PDUFA
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Vosoritide
Children with Achondroplasia
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Cerliponase alfa
Batten Disease
Approved
Approved
PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.
Valoctocogene roxaparvovec (BMN 270) - GENEr8-1
Hemophilia A
Phase 3
Phase 3
Phase 3 52-week data released January 10, 2021. All endpoints met.
BMN 307
Phenylketonuria (PKU)
Phase 1/2
Phase 1/2
Phase 1/2 commencement of dosing announced September 24, 2020.
Valoctocogene roxaparvovec (BMN 270)
Hemophilia A
CRL
CRL
CRL announced August 19, 2020.
Palynziq (Pegvaliase)
Phenylketonuria (PKU)
Approved
Approved
Approval announced May 24, 2018.
Kyndrisa
Duchenne Muscular Dystrophy (DMD)
CRL
CRL
CRL issued January 14, 2016.
Vimizim (GALNS)
(MPS IVA) Morquio A Syndrome
Approved
Approved
Approved February 14, 2014.

Latest News

  1. SAN RAFAEL, Calif., March 20, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral presentation at ENDO21, the Endocrine Society's Annual Meeting by Professor Ravi Savarirayan, M.B., B.S., M.D., clinical investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria. Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of disproportionate short stature in humans. 

    SAN RAFAEL, Calif., March 20, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral presentation at ENDO21, the Endocrine Society's Annual Meeting by Professor Ravi Savarirayan, M.B., B.S., M.D., clinical investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria. Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of disproportionate short stature in humans. 

    The data from the open-label extension presented at ENDO21 showed that children maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide.  Children who received two years of vosoritide therapy had a baseline mean AGV of 4.28 cm/year. After one year of treatment, mean AGV was 5.71 cm/year and after the second year mean AGV was 5.65 cm/year, demonstrating sustained restoration of a major portion of the growth deficit in achondroplasia through the second year of treatment. Children also had an improved height z-score, which is a measure of height relative to that of a similar population of average height.

    In the children who were crossed over from placebo to vosoritide in the open-label extension arm, similar efficacy after one year was observed compared to children who received treatment with vosoritide after one year.  Children who were crossed over to treatment had a baseline mean AGV of 3.99 cm/year.  After one year of treatment, mean AGV was 5.57 cm per year. 

    Retention of subjects on treatment was high with 93% of patients originally randomized to receive vosoritide remaining on treatment two years later.

    "This is an important extension of the original study's findings and confirm that the benefits of vosoritide are sustained during treatment through two years," said Savarirayan.  "While these two-year data are encouraging at showing a durable treatment effect, longer term follow-up will provide additional insights on improvements in body proportions and whether such improvements can potentially make a difference in certain aspects of a child's life."

    "Maintaining a consistent level of growth is important because it supports that vosoritide is addressing the root cause of achondroplasia," said Melita Irving, Clinical Geneticist at Guy's and St Thomas' NHS Foundation Trust, London, UK and investigator for the vosoritide clinical program at the Evelina London Children's Hospital. "The clinical program for vosoritide is robust and will continue to evaluate the impact on other important medical outcomes in achondroplasia through long-term evaluation. Overall, the data are encouraging, and allows us to imagine the potential for this first and only targeted precision treatment for achondroplasia."

    "We continue to take a relentless approach to advance our understanding of how vosoritide could potentially benefit children with achondroplasia, and to collect the information families and physicians will need to make informed decisions about the choice of treatment," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are encouraged by the consistency of this data with our expectations based on the known biology of CNP effects on growth, and with earlier studies, specifically with regards to durability of effect in absence of pathological advancement of bone age. We are grateful to the children, families and investigators for participating in this study and allowing the opportunity for the scientific exchange of clinical data at medical meetings like ENDO2021." 

    Vosoritide Safety

    The 2-year data demonstrated that vosoritide, administered at 15ug/kg/day was generally well tolerated with no new safety findings. The majority of adverse events (AEs) were mild and no serious adverse events were reported as study drug-related.  Injection site reactions were the most common drug-related AEs, and all were transient. There were no AEs related to disproportionate bone growth or bone pathology. No clinically significant blood pressure decreases or new safety findings were observed. 

    Regulatory Status

    In 2020, the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) accepted and validated the marketing authorization application for vosoritide for achondroplasia. The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021. In the United States, the Company has chosen to provide the 2-year outcomes from the Phase 3 extension study to the FDA as additional data to convey the vosoritide treatment effect and long-term durability. The Company believes that supplying this additional data could result in a major amendment, resetting the current PDUFA target action date out three months to November. 

    In January 2021, the Company received notice from the FDA that the NDA for vosoritide had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation.  If approved, the vosoritide NDA may qualify for a Priority Review Voucher (PRV).  A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. 

    Upon the acceptance of the regulatory submission for vosoritide, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups.  BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth and the updated 2-year data from the Phase 3 study, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. 

    Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

    Description of Phase 3 Extension Study

    This is an ongoing open-label long-term extension study to a completed pivotal, double-blind, placebo-controlled study of vosoritide in children with achondroplasia. A total of 119 children were enrolled in the extension study after completion of the pre-treatment observation period and one year of treatment in the pivotal phase 3 study, and all are receiving open-label treatment with vosoritide 15 mcg/kg daily. Vosoritide is being tested in children whose growth plates are still "open."  This is approximately 25% of people with achondroplasia. The extension study is evaluating safety, AGV, and cumulative annual height gain until participants reach final adult height.  A wide range of secondary and exploratory endpoints include anthropometric measures such as height Z-score, body and limb proportionality and joint geometry; biochemical, biomarker and radiological assessments of bone growth and health; and evaluations of health-related quality of life (HRQoL), developmental status, and functional independence. These additional endpoints address the overall impact vosoritide has on achondroplasia.

    About Achondroplasia

    Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

    More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age. This is approximately 25% of people with achondroplasia. In the U.S., Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on such website is not incorporated by reference into this press release.

    Forward-Looking Statement

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's vosoritide development program generally and specifically about the results of the extension of the Phase 3 trial, the timing of actions by regulatory authorities including the expectation of the CHMP opinion for vosoritide in Europe in June of 2021; the potential for the vosoritide NDA, if approved, to qualify for a Priority Review Voucher; and the plan to submit the second year of Phase 3 data to the FDA and the potential that this could result in a major amendment, resetting the current PDUFA date out three months to November. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: final analysis of the extension of the Phase 3 data, results and timing of current and planned preclinical studies and clinical trials of vosoritide; our ability to enroll participants into such clinical trials, our ability to record data during a global pandemic, our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the BioMarin's Securities and Exchange Commission (SEC) filings, including, without limitation BioMarin's Annual Report on Form 10-K for the year ended December 31, 2020, as such factors may be updated by any subsequent reports. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contacts:

    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558 

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-oral-presentation-at-endo2021-the-endocrine-societys-annual-meeting-with-data-demonstrating-2-years-of-treatment-benefit-in-children-with-achondroplasia-treated-with-vosoritide-301252378.html

    SOURCE BioMarin Pharmaceutical Inc.

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  2. SAN RAFAEL, Calif., March 8, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. The FDA granted RMAT designation based on the potential of the valoctocogene roxaparvovec clinical data to address the unmet medical need within this population. 

    RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017, allowing early, close, and frequent interactions with the FDA. One additional feature of the RMAT program is that sponsors of products that have been granted RMAT designation and which receive accelerated approval may be able to fulfill the post-approval requirements from clinical evidence obtained from sources other than the traditional confirmatory clinical trials.

    The RMAT designation comes coincidently during Bleeding Disorders Awareness Month initiated by the National Hemophilia Foundation to celebrate and honor the bleeding disorders community.

    "We are encouraged that the FDA granted RMAT Designation to valoctocogene roxaparvovec.  This designation confirms our belief in this treatment's potential to address unmet medical needs for people with hemophilia A at this time," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We look forward to continuing a productive dialogue with the FDA around the RMAT designation, which provides options for the Agency to leverage data post approval, while also recognizing the agency's initial request to see two years of data from the Phase 3 study to evaluate the safety and efficacy of this investigational treatment option that could potentially provide a transformational treatment choice for the hemophilia community."

    "During Bleeding Disorders Awareness Month, we applaud the FDA for its efforts to recognize the potential of cell and gene therapies to help people with hemophilia who have medical needs not currently addressed," said Leonard A. Valentino, MD, President & CEO of the National Hemophilia Foundation (NHF). "Established under the 21st Century Cures Act, RMAT designation has the potential to provide more treatment choices to people with bleeding disorders at a faster pace, which benefits the whole community. The FDA's RMAT designation is a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the potential to address unmet needs," said the Alliance for Regenerative Medicine (ARM). "As the global voice of the regenerative medicine sector, ARM played a critical role in the creation of this pathway. The FDA has granted more than 50 RMAT designations to investigational products and in February approved an RMAT-designated product for the first time, illustrating the agency's commitment to advancing the development of regenerative medicines." 

    Regulatory Status

    In the Complete Response Letter of August 18, 2020 to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec, the FDA recommended that the Company complete the Phase 3 GENEr8-1 study and submit two-year follow-up safety and efficacy data on all study participants. The Company plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021. In the EU, BioMarin is targeting submission of the Marketing Authorization Application (MAA) with these results to the EMA in the second quarter of 2021 pending confirmation in planned pre-submission meetings.

    In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. 

    Robust Clinical Program

    BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company recently began enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A. The Company is running a Phase 1/2 Study with the 6e13kg/vg dose of valoctocogene roxaparvovec in approximately 10 participants with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors. 

    About Hemophilia A

    People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

    Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Forward Looking Statements

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about the development of BioMarin's valoctocogene roxaparvovec program generally, the RMAT designation, the Company's plans to meet with the FDA to review the 2-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021, BioMarin targeting submission of the MAA with these results to the EMA in the second quarter of 2021; the potential of valoctocogene roxaparvovec to address the unmet medical need within this hemophilia A  population.  These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of data from the continuation of these trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture the product candidate for the preclinical and clinical trials;  and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including without limitation, BioMarin's Annual Report on Form 10-K for the year ended December 31, 2020 as such factors may be updated by any subsequent reports. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contacts:



    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-fda-regenerative-medicine-advanced-therapy-rmat-designation-granted-to-valoctocogene-roxaparvovec-investigational-gene-therapy-for-hemophilia-a-301242135.html

    SOURCE BioMarin Pharmaceutical Inc.

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  3. SAN RAFAEL, Calif., March 3, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial where all children receive active treatment. Children in this study will have completed a minimum three- or six-month baseline…

    SAN RAFAEL, Calif., March 3, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial where all children receive active treatment. Children in this study will have completed a minimum three- or six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on growth. The company also plans to augment the height data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.

    There are currently no approved pharmacological treatments for achondroplasia, with existing treatments mainly limited to surgical interventions to address a variety of symptoms. This treatment gap presents a significant unmet need. Vosoritide is an investigational therapy that seeks to directly target the root cause of achondroplasia by interrupting the pathway that slows bone growth due to the causative mutation in achondroplasia. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, such as foramen magnum compression, sleep apnea, and spinal stenosis. Some of these complications can result in the need for invasive surgeries. In addition, studies show increased mortality at every age.

    "This milestone is an important building block of a comprehensive clinical program that is methodically and responsibly studying this potential first pharmacological treatment choice for achondroplasia with the goal of further understanding the safety and efficacy in the youngest children," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "In this trial, we are studying the effects of vosoritide during the most productive time of growth. We are grateful to the children and families enrolled in this placebo-controlled study and are committed to the long-term follow up of the children in these studies."

    "This is an exciting milestone for children and families, who are interested in a treatment choice to address the basic cause of the irregular bone growth seen in achondroplasia. It represents a potential medical breakthrough that would be the first non-surgical treatment for children with achondroplasia," said John A. Phillips, III, M.D., Vanderbilt University Medical Center (David T Karzon Professor of Pediatrics) and investigator for the vosoritide clinical program. "As a treating physician, I see an urgent demand from families for a treatment option that addresses bone growth and potentially the serious complications associated with achondroplasia, especially during infancy." 

    "This milestone is a giant step towards improving the quality of medical care and options available to individuals with achondroplasia and to their families," said Munira Shamim, co-founder of Growing Stronger. Many families are eagerly awaiting a drug treatment option that could possibly decrease the risk of health issues associated with achondroplasia and increase the quality of life. We would like to recognize the committed families and participants in the placebo-controlled studies for collaborating with dedicated scientists to further scientific learning that can potentially change the lives of thousands of families."

    Regulatory Status

    In 2020, the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) accepted and validated the marketing authorization application for vosoritide for achondroplasia. The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021. In the United States, the Company has chosen to provide the 2-year outcomes from the Phase 3 extension study to the FDA as additional data to convey the vosoritide treatment effect and long-term durability. The Company believes that supplying this additional data could result in a major amendment, resetting the current PDUFA target action date out three months to November. 

    In January 2021, the Company received notice from the FDA that the NDA for vosoritide had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation. If approved, the vosoritide NDA may qualify for a Priority Review Voucher (PRV). A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. 

    Upon the acceptance of the regulatory submission for vosoritide, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth and the updated 2-year data from the Phase 3 study, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. 

    Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

    Description of Phase 2 Study in Infants and Young Children Ages 0 to 5 Years

    This is a Phase 2 randomized, placebo-controlled study of vosoritide. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will have completed a three-to-six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height. The company also plans to augment the height data with other analyses of effects on growth and assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries. 

    About Achondroplasia

    Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

    More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.  In the U.S., Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on such website is not incorporated by reference into this press release.

    Forward-Looking Statement

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's vosoritide program generally; the potential benefits of vosoritide for infants and young children; the continued clinical development of vosoritide; the timing, design and conduct of the planned Phase 2 study in infants and young children and the expectation that topline results from this Phase 2 study will be released in mid-2022; the timing, design and conduct of other ongoing and possible future studies of vosoritide; the expected results of such studies, the ability to use the primary objectives of the Phase 2 study to support the use of vosoritide in infants and young children; the timing of actions by regulatory authorities including the expectation of the CHMP opinion for vosoritide in Europe in June of 2021; the potential for the vosoritide NDA, if approved, to qualify for a Priority Review Voucher; and the plan to submit the second year of Phase 3 data to the FDA and the potential that this could result in a major amendment, resetting the current PDUFA date out three months to November. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of vosoritide; our ability to enroll participants into such clinical trials, our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings, including, without limitation, BioMarin's Annual Report on Form 10-K for the year ended December 31, 2020 as such factors may be updated by any subsequent reports. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contact:



    Investors:

    Media:

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-completes-full-enrollment-in-phase-2-study-of-vosoritide-for-treatment-of-infants-and-young-children-with-achondroplasia-301239664.html

    SOURCE BioMarin Pharmaceutical Inc.

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  4. SAN RAFAEL, Calif., Feb. 25, 2021 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

    Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)


    Three Months Ended December 31,


    Twelve Months Ended December 31,


    2020


    2019


    % Change


    2020


    2019


    % Change













    Total Revenues

    $

    452.1



    $

    454.4



    (1)

    %


    $

    1,860.5



    $

    1,704.0



    9

    %













    Net Product Revenues Marketed by BioMarin (1)

    435.8



    412.7



    6

    %


    1,675.8



    1,563.2



    7

    %













    Vimizim Net Product Revenues

    142.5



    132.3



    8

    %


    544.4



    544.3



    %

    Kuvan Net Product Revenues

    89.0



    122.6



    (27)

    %


    457.7



    463.4



    (1)

    %

    Naglazyme Net Product Revenues

    119.7



    94.8



    26

    %


    391.3



    374.3



    5

    %

    Palynziq Net Product Revenues

    49.6



    31.7



    56

    %


    171.0



    86.9



    97

    %

    Brineura Net Product Revenues

    35.0



    25.2



    39

    %


    110.2



    72.0



    53

    %













    Aldurazyme Net Product Revenues

    1.2



    23.9



    (95)

    %


    130.1



    97.8

    SAN RAFAEL, Calif., Feb. 25, 2021 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

    Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)



    Three Months Ended December 31,



    Twelve Months Ended December 31,



    2020



    2019



    % Change



    2020



    2019



    % Change

























    Total Revenues

    $

    452.1





    $

    454.4





    (1)

    %



    $

    1,860.5





    $

    1,704.0





    9

    %

























    Net Product Revenues Marketed by BioMarin (1)

    435.8





    412.7





    6

    %



    1,675.8





    1,563.2





    7

    %

























    Vimizim Net Product Revenues

    142.5





    132.3





    8

    %



    544.4





    544.3





    %

    Kuvan Net Product Revenues

    89.0





    122.6





    (27)

    %



    457.7





    463.4





    (1)

    %

    Naglazyme Net Product Revenues

    119.7





    94.8





    26

    %



    391.3





    374.3





    5

    %

    Palynziq Net Product Revenues

    49.6





    31.7





    56

    %



    171.0





    86.9





    97

    %

    Brineura Net Product Revenues

    35.0





    25.2





    39

    %



    110.2





    72.0





    53

    %

























    Aldurazyme Net Product Revenues

    1.2





    23.9





    (95)

    %



    130.1





    97.8





    33

    %

























    GAAP Net Income (Loss)

    $

    22.1





    $

    15.0









    $

    859.1





    $

    (23.8)







    GAAP Net Income (Loss) per Share – Basic

    $

    0.12





    $

    0.08









    $

    4.75





    $

    (0.13)







    GAAP Net Income (Loss) per Share – Diluted

    $

    0.12





    $

    0.08









    $

    4.53





    $

    (0.13)







    Non-GAAP Income (2)

    $

    39.5





    $

    46.4









    $

    312.2





    $

    166.6







     



    December 31,

    2020



    December 31,

    2019



    Cash, cash equivalents and investments

    $

    1,350.9





    $

    1,165.8



























    (1)

    Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura and Firdapse, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP). Sanofi Genzyme (Genzyme) is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties. Refer to page 8 for a table showing Net Product Revenues by product. In January 2020, BioMarin divested the Firdapse assets to a third party in a sale transaction. The sale is reflected in the Company's consolidated financial statements for the twelve months ending December 31, 2020; as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

    (2)

    Non-GAAP Income is defined by the Company as reported GAAP Net Income/Loss, excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items. Refer to Non-GAAP Information beginning on page 10 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP.

    BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) (BioMarin or the Company) today announced financial results for the fourth quarter and full year ended December 31, 2020.

    Net Product Revenues for the fourth quarter of 2020 were essentially flat as compared to the fourth quarter of 2019. The change in Net Product Revenues was primarily attributed to the following:

    • Kuvan Net Product Revenues decreased by $33.6 million, primarily due to the U.S. loss of market exclusivity in October 2020 resulting from generic competition; and
    • Aldurazyme Net Product Revenues decreased by $22.7 million due to timing of product fulfillment to Genzyme. Aldurazyme is marketed by Genzyme and BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Genzyme. Revenues for the fourth quarter of 2020 were comparatively lower than 2019 due to such timing. Based on data provided to us by Genzyme, patients receiving commercial Aldurazyme increased by 10% during 2020; partially offset by
    • Naglazyme and Vimizim Net Product Revenues increased by an aggregate of $35.1 million primarily due to timing of sales in the Middle East and Latin America;
    • Palynziq Net Product Revenues increased by $17.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy; and
    • Brineura Net Product Revenues increased by $9.8 million driven by growth in the number of patients in all regions.

    The increase in GAAP Net Income for the fourth quarter of 2020, compared to the same period in 2019 was primarily due to the following:

    • decreased research and development (R&D) expense of $16.1 million primarily due to lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs and decreased tralesinidase alfa costs as the program was licensed to a third-party in 2019; and
    • an increase in the benefit from income taxes of $37.5 million primarily due to the change in the jurisdictional mix of earnings and the related tax impact from the completion of an intra-entity transfer of certain intellectual property rights to an Irish subsidiary where the Company's ex-US regional headquarters are located during the third quarter of 2020; partially offset by
    • an increase in Cost of Sales of $30.2 million primarily due to inventory reserves and higher sales volumes of products with lower margins.

    Non-GAAP Income for the fourth quarter of 2020 decreased to $39.5 million, compared to Non-GAAP Income of $46.4 million for the same period in 2019. The decrease in Non-GAAP Income for the quarter, compared to the same period in 2019, was primarily attributed to lower gross profits and higher SG&A expenses, partially offset by lower R&D expenses.

    As of December 31, 2020, BioMarin had cash, cash equivalents and investments totaling $1.35 billion, which includes net proceeds of $535.8 million from the Company's May 2020 convertible debt offering, as compared to $1.17 billion as of December 31, 2019. On October 15, 2020, the Company's 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.

    Commenting on full-year 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "Despite the impact in 2020 from the COVID-19 pandemic and a delay in the potential approval of valoctocogene roxaparvovec for severe hemophilia A, demand for our current product portfolio continued to drive steady revenue growth and expansion of our pipeline.  Excluding contributions from Kuvan, for which a generic became available during 2020, total revenues grew 13% in 2020, and generated $85 million of positive operating cash flows for the full year, underscoring the essential nature of our medicines." 

    Mr. Bienaimé continued, "The most recent Phase 3 data updates from our latest-stage development programs in achondroplasia and severe hemophilia A demonstrated significant efficacy.  In the largest gene therapy trial ever conducted for the treatment of severe hemophilia A, we were pleased that valoctocogene roxaparvovec was the first in hemophilia A to demonstrate statistically significant evidence of annualized bleed rate superiority over standard of care recombinant FVIII. Based on these results, we are very encouraged that one infusion of valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A.  We are targeting submission of the one-year Phase 3 results to the European Medicines Agency in the second quarter of 2021 and planning to dialog with the FDA to align on steps to obtain approval in the United States."

    "Also in 2021, we look forward to the potential approval of vosoritide, which would be the first pharmacological treatment to address the underlying cause of achondroplasia, the most common form of dwarfism.  We announced in the fourth quarter of 2020 that vosoritide demonstrated sustained growth effects for over two years in children with achondroplasia participating in our Phase 3 extension study.  In addition to the large, Phase 3 program currently in the extension phase, we have built a multi-pronged dossier of additional studies to support our understanding of the unmet medical need for children with achondroplasia and the effects of vosoritide in this condition. In addition to the highly statistically significant placebo-controlled Phase 3 results, the program includes the long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years. Many families are keen to seek early treatment for their children so we are hopeful that, if approved, vosoritide will become available later in 2021 upon potential approvals."

    2021 Full-Year Financial Guidance (in millions, except %)

    Item



    2021 Guidance *

    Total Revenues



    $1,750



    to



    $1,850

    Vimizim Net Product Revenues



    $570



    to



    $610

    Kuvan Net Product Revenues



    $250



    to



    $290

    Naglazyme Net Product Revenues



    $365



    to



    $395

    Palynziq Net Product Revenues



    $210



    to



    $250

    Brineura Net Product Revenues



    $120



    to



    $140















    Cost of Sales (% of Total Revenues)



    23%



    to



    25%

    Research and Development Expense



    $645



    to



    $695

    Selling, General and Administrative Expense



    $725



    to



    $775















    GAAP Net Loss



    ($130)



    to



    ($80)

    Non-GAAP Income (1)



    $170



    to



    $220



















    *2021 Guidance takes into consideration ongoing expected impact from the COVID-19 pandemic in 2021 assuming consistent trends experienced during 2020.





    (1)

    All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income/Loss. Refer to Non-GAAP Information beginning on page 10 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the corresponding GAAP reported information.

    Key Program Highlights

    • Valoctocogene roxaparvovec gene therapy for severe hemophilia A: On January 10, 2021, the Company announced positive top-line, one-year data results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. Data from the study in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) showed that a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% compared with prior treatment with prophylactic FVIII infusions. These results were from a pre-specified group of participants in a non-interventional prospective baseline observational study (rollover population; N=112) with a median follow-up of 60.1 weeks after dosing with valoctocogene roxaparvovec. 80% of the rollover participants were bleed-free starting at week five after treatment.

    Additionally, at the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion treatment rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2 (SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year.

    Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII usage in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.0001).

    In the U.S., the FDA recommended that the Company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. The Company plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021. BioMarin is targeting submission of the Marketing Authorization Application (MAA) with these results to the EMA in the second quarter of 2021 pending confirmation in presubmission meetings.

    • Vosoritide for children with achondroplasia: On December 21, 2020 the Company announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment. An analysis, comparing all children randomized and treated with vosoritide for two years (n=52) to all children from the run-in study who were randomized to receive placebo with an untreated observation period of two years (n=38), showed improvement in one-year height change in the treated group relative to the untreated group that was similar in the second year of treatment, 1.79 cm, as in the first year of treatment, 1.73 cm. The cumulative height gain over the 2-year treatment period was 3.52 cm more than the untreated children.

    In 2020, marketing applications for vosoritide were validated and accepted by EMA and FDA, respectively. The CHMP opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021.

    In January 2021, the Company received notice from FDA that the NDA for vosoritide had been granted Priority Review Designation. Under this designation, the vosoritide NDA may qualify for a Priority Review Voucher (PRV) upon approval.  A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

    • Palynziq for PKU: On October 7, 2020 the Company announced that the FDA approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of Palynziq (pegvaliase-pqpz) Injection for treatment of adults with PKU to 60 mg daily. Previously, the maximum dose was 40 mg daily. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.

    Palynziq is indicated to reduce blood Phe concentrations in adults with PKU, who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

    • BMN 307 gene therapy product candidate for PKU: The Company announced that it plans to dose escalate in PHEarless, the Phase 1/2 study of BMN 307 based on encouraging Phe lowering and safety signals observed in study participants who were treated with the lowest dose. Both the FDA and EMA granted BMN 307 Orphan Drug Status. Additionally, the FDA has granted Fast Track status to BMN 307. Product for use in the Phase 1/2 study was made at commercial scale from BioMarin's award-winning gene therapy manufacturing facility.
    • BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): IND-enabling studies are ongoing with BMN 331, BioMarin's third gene therapy candidate, for the treatment of HAE. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331.
    • DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
    • BMN 255 for a subset of chronic renal disease: On January 11, 2021 the Company announced that it filed an IND in 2020 for BMN 255, a small molecule for a subset of chronic renal disease. BMN 255 was driven by genetic discoveries for both mechanism and for identifying individuals for treatment.
    • BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an oligonucleotide therapy that has demonstrated a high-level of protein expression in experimental animals possessing skippable dystrophic mutations and at doses that are promising in regard to safety. The Company intends to determine timing of a potential IND filing at the end of the year based on results of ongoing IND-enabling studies.

    BioMarin will host a conference call and webcast to discuss fourth quarter and full-year 2020 financial results today, Thursday, February 25, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

    U.S./Canada Dial-in Number: 866.502.9859

    Replay Dial-in Number: 855.859.2056

    International Dial-in Number: 574.990.1362

    Replay International Dial-in Number: 404.537.3406

    Conference ID: 6488682

    Conference ID: 6488682

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of several commercial therapies and multiple clinical and preclinical product candidates.

    For additional information, please visit www.biomarin.com.

    Forward-Looking Statements

    This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expectations of Total Revenues, Net Product Revenues, Research and Development Expense, Selling, General and Administrative Expense, Cost of Sales, GAAP Net Loss, Non-GAAP Income, and other specified income statement guidance for the full-year 2021; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) BioMarin's plan to submit complete one-year Phase 3 data for valoctocogene roxaparvovec to the EMA in the second quarter of 2021, (ii) BioMarin's plan to resubmit its MAA for valoctocogene roxaparvovec to the EMA in the second quarter of 2021 (iii) BioMarin's plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021, (iv) that the CHMP opinion for vosoritide is expected in Europe in the second half of 2021; and (v) the target PDUFA action date with respect to vosoritide of August 20, 2021; the potential approval and commercialization of BioMarin's product candidates, including vosoritide for the treatment of achondroplasia and valoctocogene roxaparvovec for the treatment of severe hemophilia A, including timing of such approval decisions; and the expected benefits and availability of BioMarin's product candidates, including (i) that valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A and (ii) BioMarin's hope that, if approved, vosoritide will become available later in 2021.

    These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products, including BioMarin's projected impact of the COVID-19 pandemic on its global revenue sources, including due to demand interruptions such as missed patient infusions and delayed treatment starts for new patients; results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on (i) BioMarin's ability to continue such preclinical studies and clinical trials and (ii) the timing of such preclinical studies and clinical trials, and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning each of the described products and product candidates, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the market for each of these products; actual sales of BioMarin's commercial products and the impact that the COVID-19 pandemic may have on such sales; the introduction of generic versions of BioMarin's commercial products, in particular generic versions of Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

    BioMarin®, Brineura®, Kuvan®, Naglazyme®, Palynziq® and Vimizim® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    December 31, 2020 and December 31, 2019

    (In thousands of U.S. dollars, except per share amounts)





    December 31,

    2020



    December 31,

    2019(1)

    ASSETS

    (unaudited)





    Current assets:







    Cash and cash equivalents

    $

    649,158





    $

    437,446



    Short-term investments

    416,228





    316,361



    Accounts receivable, net

    448,351





    377,404



    Inventory

    698,548





    680,275



    Other current assets

    129,934





    130,657



    Total current assets

    2,342,219





    1,942,143



    Noncurrent assets:







    Long-term investments

    285,473





    411,978



    Property, plant and equipment, net

    1,032,471





    1,010,868



    Intangible assets, net

    417,271





    456,580



    Goodwill

    196,199





    197,039



    Deferred tax assets

    1,432,150





    549,422



    Other assets

    142,237





    122,009



    Total assets

    $

    5,848,020





    $

    4,690,039



    LIABILITIES AND STOCKHOLDERS' EQUITY







    Current liabilities:







    Accounts payable and accrued liabilities

    $

    492,548





    $

    570,621



    Short-term convertible debt, net





    361,882



    Total current liabilities

    492,548





    932,503



    Noncurrent liabilities:







    Long-term convertible debt, net

    1,075,145





    486,238



    Long-term contingent consideration

    60,130





    50,793



    Other long-term liabilities

    114,195





    98,124



    Total liabilities

    $

    1,742,018





    $

    1,567,658



    Stockholders' equity:







    Common stock, $0.001 par value: 500,000,000 shares authorized; 181,740,999 and 179,838,114 shares issued and outstanding, respectively.

    182





    180



    Additional paid-in capital

    4,993,407





    4,832,707



    Company common stock held by Nonqualified Deferred Compensation Plan

    (9,839)





    (9,961)



    Accumulated other comprehensive income

    (16,139)





    20,164



    Accumulated deficit

    (861,609)





    (1,720,709)



    Total stockholders' equity

    4,106,002





    3,122,381



    Total liabilities and stockholders' equity

    $

    5,848,020





    $

    4,690,039















    (1)

    December 31, 2019 balances were derived from the audited Consolidated Financial Statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2019, filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020.



     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    Three and Twelve Months Ended December 31, 2020 and 2019

    (In thousands of U.S. dollars, except per share amounts)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    2020



    2019 (1)



    (unaudited)



    (unaudited)



    (unaudited)





    REVENUES:















    Net product revenues

    $

    437,045





    $

    436,585





    $

    1,805,861





    $

    1,661,043



    Royalty and other revenues

    15,072





    17,858





    54,594





    43,005



    Total net revenues

    452,117





    454,443





    1,860,455





    1,704,048



    OPERATING EXPENSES:















    Cost of sales

    126,138





    95,899





    524,272





    359,466



    Research and development

    156,667





    172,812





    628,116





    715,007



    Selling, general and administrative

    195,512





    187,900





    737,669





    680,924



    Intangible asset amortization and contingent consideration

    18,640





    16,994





    66,658





    74,108



    Gain on sale of nonfinancial assets





    (10,000)





    (59,495)





    (25,000)



    Total operating expenses

    496,957





    463,605





    1,897,220





    1,804,505



    LOSS FROM OPERATIONS

    (44,840)





    (9,162)





    (36,765)





    (100,457)



















    Equity in the income (loss) of BioMarin/Genzyme LLC

    1,071





    193





    (6)





    (587)



    Interest income

    3,071





    5,211





    16,610





    22,748



    Interest expense

    (4,749)





    (6,930)





    (29,309)





    (23,460)



    Other income, net

    5,262





    907





    7,148





    6,945



    LOSS BEFORE INCOME TAXES

    (40,185)





    (9,781)





    (42,322)





    (94,811)



    Benefit from income taxes

    (62,284)





    (24,805)





    (901,422)





    (70,963)



    NET INCOME (LOSS)

    22,099





    15,024





    859,100





    (23,848)



    NET INCOME (LOSS) PER SHARE, BASIC

    $

    0.12





    $

    0.08





    $

    4.75





    $

    (0.13)



    NET INCOME (LOSS) PER SHARE, DILUTED

    $

    0.12





    $

    0.08





    $

    4.53





    $

    (0.13)



    Weighted average common shares outstanding, basic

    181,435





    179,531





    180,804





    179,039



    Weighted average common shares outstanding, diluted

    184,476





    182,412





    191,678





    179,039







    (1)

    December 31, 2019 totals were derived from the audited Consolidated Financial Statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on February 27, 2020.



    The following table presents Net Product Revenues by Product:

    Net Product Revenues by Product

    (In millions of U.S. dollars)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    % Change



    2020



    2019 (1)



    % Change



    (unaudited)



    (unaudited)







    (unaudited)









    PKU franchise

    $

    138.6





    $

    154.3





    (10)

    %



    $

    628.7





    $

    550.3





    14

    %

    Vimizim

    142.5





    132.3





    8

    %



    544.4





    544.3





    %

    Naglazyme

    119.7





    94.8





    26

    %



    391.3





    374.3





    5

    %

    Brineura

    35.0





    25.2





    39

    %



    110.2





    72.0





    53

    %

    Firdapse (2)





    6.1





    (100)

    %



    1.2





    22.3





    (95)

    %

    Net Product Revenues Marketed

           by BioMarin

    435.8





    412.7









    1,675.8





    1,563.2







    Aldurazyme Net Product Revenues

          Marketed by Genzyme

    1.2





    23.9





    (95)

    %



    130.1





    97.8





    33

    %

    Total Net Product Revenues

    $

    437.0





    $

    436.6









    $

    1,805.9





    $

    1,661.0











    (1)

    December 31, 2019 totals were derived from the audited Consolidated Financial Statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on February 27, 2020.

    (2)

    In January 2020, BioMarin divested the Firdapse assets to a third party in a sale transaction. The sale is reflected in the Company's consolidated financial statements for the twelve months ended months ending December 31, 2020; and as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

    The following table presents Net Product Revenues for the PKU Franchise by Product:

    Net Product Revenues by Product for the PKU Franchise

    (In millions of U.S. dollars)

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    % Change



    2020



    2019 (1)



    % Change



    (unaudited)



    (unaudited)







    (unaudited)









    Kuvan

    $

    89.0





    122.6





    (27)

    %



    $

    457.7





    463.4





    (1)

    %

    Palynziq

    49.6





    31.7





    56

    %



    171.0





    86.9





    97

    %

    Total PKU franchise

    $

    138.6





    $

    154.3





    (10)

    %



    $

    628.7





    $

    550.3





    14

    %

















































    (1)

    December 31, 2019 totals were derived from the audited Consolidated Financial Statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on February 27, 2020.



    Non-GAAP Information

    The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income is defined by the Company as GAAP Net Income/Loss excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Income/Loss for each of the periods presented. In this regard, Non-GAAP Income and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures.

    BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income and its components are important internal measurements for BioMarin, the Company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the Company's results from period to period and to its forward-looking guidance, and to identify operating trends in the Company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure.

    Non-GAAP Income and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

    The following table presents the reconciliation of GAAP Net Income (Loss) to Non-GAAP Income:

    Reconciliation of GAAP Net Income (Loss) to Non-GAAP Income

    (In millions of U.S. dollars)

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended December 31,



    Guidance

    Year Ending



    2020



    2019



    2020



    2019



    December 31, 2021

























    GAAP Net Income (Loss)

    $

    22.1





    $

    15.0





    $

    859.1





    $

    (23.8)





    $

    (130.0)



    $

    (80.0)



























    Interest expense, net

    1.7





    1.7





    12.7





    0.7





    11.0



    11.0



    Benefit from income taxes

    (62.3)





    (24.8)





    (901.4)





    (71.0)





    (16.0)



    (16.0)



    Depreciation expense

    11.9





    9.5





    43.0





    51.8





    48.0



    48.0



    Amortization expense

    15.4





    16.3





    62.2





    53.5





    62.0



    62.0



    Stock-based compensation expense

    47.5





    38.0





    189.7





    159.8





    186.0



    186.0



    Contingent consideration expense

    3.2





    0.7





    4.5





    20.6





    9.0



    9.0



    Provision for inventory reserve, net (1)









    75.6













    Gain on sale of nonfinancial assets





    (10.0)





    (59.5)





    (25.0)









    Licensed In-Process R&D (2)









    26.3













    Non-GAAP Income

    $

    39.5





    $

    46.4





    $

    312.2





    $

    166.6





    $

    170.0



    $

    220.0







    (1)

    Represents a $81.2 million charge related to pre-launch valoctocogene roxaparvovec inventory, net of stock-based compensation, as a result of the unexpected delays in anticipated regulatory approvals.

    (2)

    Represents the upfront license fee paid to a third party and recognized as R&D expense in the second quarter of 2020.

    The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.

    Reconciliation of Certain GAAP Reported Information to Non-GAAP Information

    (In millions of U.S. dollars)

    (unaudited)





    Three months ended December 31,



    2020



    2019







    Adjustments











    Adjustments







































    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP



    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP

    Cost of sales

    $

    126.1





    $





    $

    (5.9)





    $

    120.2





    $

    95.9





    $





    $

    (3.5)





    $

    92.4



    Research and development

    156.7





    (6.7)





    (16.5)





    133.5





    172.8





    (3.8)





    (13.5)





    155.5



    Selling, general and administrative

    195.5





    (5.2)





    (25.1)





    165.2





    187.9





    (5.7)





    (21.0)





    161.2



    Intangible asset amortization and contingent consideration

    18.6





    (15.4)





    (3.2)









    17.0





    (16.3)





    (0.7)







    Gain on sale of nonfinancial assets

















    (10.0)









    10.0







    Interest expense, net

    (1.7)





    1.7













    (1.7)





    1.7











    Benefit from income taxes

    (62.3)





    62.3













    (24.8)





    24.8











    GAAP Net Income /Non-GAAP Income

    $

    22.1





    $

    (33.3)





    $

    50.7





    $

    39.5





    $

    15.0





    $

    2.7





    $

    28.7





    $

    46.4



































     



    Twelve months ended December 31,



    2020



    2019







    Adjustments











    Adjustments







































    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP



    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP

    Cost of sales

    $

    524.3





    $





    $

    (101.9)





    $

    422.4





    $

    359.5





    $





    (16.1)





    $

    343.4



    Research and development

    628.1





    (21.9)





    (88.2)





    518.0





    715.0





    (26.9)





    (56.6)





    631.5



    Selling, general and administrative

    737.7





    (21.1)





    (101.5)





    615.1





    680.9





    (24.9)





    (87.1)





    568.9



    Intangible asset amortization and contingent consideration

    66.7





    (62.2)





    (4.5)









    74.1





    (53.5)





    (20.6)







    Gain on sale of nonfinancial assets

    (59.5)









    59.5









    (25.0)









    25.0







    Interest expense, net

    (12.7)





    12.7













    (0.7)





    0.7











    Benefit from income taxes

    (901.4)





    901.4













    (71.0)





    71.0











    GAAP Net Income (Loss)/Non-GAAP Income

    859.1





    (783.5)





    236.6





    312.2





    (23.8)





    35.0





    155.4





    166.6



     

    Contact:





    Investors:



    Media:

    Traci McCarty



    Debra Charlesworth

    BioMarin Pharmaceutical Inc.



    BioMarin Pharmaceutical Inc.

    (415) 455-7558



    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-fourth-quarter-and-record-full-year-2020-financial-results-and-corporate-updates-301236040.html

    SOURCE BioMarin Pharmaceutical Inc.

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  5. SAN RAFAEL, Calif., Feb. 24, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in four upcoming virtual conferences.  An audio webcast of the presentations will be available live. You can access the webcast at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. 

    For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

     

    Contacts:



    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558 

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-participate-in-four-upcoming-virtual-investor-conferences-301234450.html

    SOURCE BioMarin Pharmaceutical Inc.

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