BMRN BioMarin Pharmaceutical Inc.

77.39
0  0%
Previous Close 77.39
Open
52 Week Low 62.88
52 Week High 131.945
Market Cap $14,034,645,312
Shares 181,349,597
Float 158,527,781
Enterprise Value $14,108,962,311
Volume 2,341
Av. Daily Volume 2,650,646
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Upcoming Catalysts

Drug Stage Catalyst Date
Cerliponase alfa
Batten Disease
Approved
Approved
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Valoctocogene roxaparvovec (BMN 270) - GENEr8-1
Hemophilia A
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
BMN 307
Phenylketonuria (PKU)
Phase 1/2
Phase 1/2
Phase 1/2 commencement of dosing announced September 24, 2020.
Vosoritide
Achondroplasia
NDA Filing
NDA Filing
NDA filing announced August 20, 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., Sept. 24, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU.  BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU. 

    SAN RAFAEL, Calif., Sept. 24, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU.  BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU. 

    BioMarin will conduct this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval.  BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program. 

    "More than 70 years ago, the first child was treated for PKU in the United Kingdom at Birmingham Women's and Children's Hospital.  Today, we continue to make strides in PKU treatment through the clinical study of a gene therapy for PKU," said Tarekegn G. Hiwot at University Hospitals Birmingham NHS Foundation Trust and principal investigator for the PHEARLESS study.  "There is a tremendous unmet need for PKU patients.  As a treating physician, it is important to me to be involved in clinical research to evaluate innovative therapies that have the potential to change the treatment paradigm in PKU for good."

    "BioMarin has been committed to the PKU community for more than 15 years and remains dedicated to the research and development of innovative therapies to advance the standard of care for people with PKU," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.  "Building upon our experience of delivering two approved PKU therapies to the PKU community, BMN 307 gene therapy combines BioMarin's leadership in the development of PKU therapies with our expertise in gene therapy development and manufacturing."

    "PKU is a serious condition and many individuals struggle to manage their disorder on a daily basis.  BioMarin is a pioneer in PKU treatments delivering the first two drug therapies to individuals with PKU.  We applaud their unwavering commitment to drive research to bring a third treatment to the PKU community and for their substantial contributions to the overall body of scientific knowledge in PKU that they continue to make,"  said Christine S. Brown, MS, Executive Director, National PKU Alliance.  "We are encouraged by BioMarin's efforts to develop a gene therapy that brings together their experience in PKU drug development, gene therapy development and gene therapy manufacturing. "

    PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

    Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.  The Company is actively preparing regulatory submissions to open additional clinical sites in other countries. 

    BMN 307 Clinical Program

    BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU.  The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated.  In addition, BioMarin is sponsoring an observational study, PHENOM, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

    BioMarin's 15-Plus Year Commitment to PKU Research

    For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients.  BioMarin has treated approximately 7,000 PKU patients around the world.  The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development.  BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies.  BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.  

    About Gene Therapy

    Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

    Gene Therapy Manufacturing

    BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California that was cGMP certified by the EMA in Q2 2020.  This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

    Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency.  The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future. 

    About Phenylketonuria

    PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels.  Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

    To learn more about PKU and PAH deficiency, please visit www.PKU.com. Information on this website is not incorporated by reference into this press release.

    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 BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, the impact of using material manufactured at commercial scale in a clinical trial, and the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307. 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 BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; 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 June 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® 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-pioneer-in-phenylketonuria-pku-and-gene-therapy-doses-first-participant-in-global-phearless-phase-12-study-of-bmn-307-gene-therapy-301137556.html

    SOURCE BioMarin Pharmaceutical Inc.

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  2. SAN RAFAEL, Calif., Sept. 15, 2020 /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-301128234.html

    SOURCE BioMarin Pharmaceutical Inc.

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  3. SAN RAFAEL, Calif., Sept. 11, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that Lynda Polgreen, MD, MS, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA will present data from the randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET. Achondroplasia is the most common form of disproportionate short stature in humans.

    SAN RAFAEL, Calif., Sept. 11, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that Lynda Polgreen, MD, MS, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA will present data from the randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET. Achondroplasia is the most common form of disproportionate short stature in humans.

    "We are pleased to share this phase 3 clinical data with the research community at this important conference and contribute to the growing body of scientific information about vosoritide, an investigational therapeutic to address the underlying molecular pathology in children with achondroplasia," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "This study is part of a robust clinical program, and we are grateful to the participating children, families and physicians."

    Regulatory Status

    BioMarin has previously announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application and that a New Drug Application (NDA) for vosoritide has been submitted to the U.S. Food and Drug Administration (FDA). 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 Study

    The global Phase 3 study was a randomized, double-blind, placebo-controlled study of vosoritide in 121 children with achondroplasia aged 5 to 14 for 52 weeks. (The enrollment age criteria were 5 to 18 per the study protocol). Vosoritide is being tested in children whose growth plates are still open. This is approximately 25% of people with achondroplasia. Children in the Phase 3 study completed a minimum six-month baseline study to determine their baseline growth velocity prior to entering the Phase 3 study. The primary endpoint of the study was the change in growth velocity from baseline over one year in children treated with vosoritide compared to placebo. Children in the study will continue to be evaluated in an ongoing open-label extension study where all study participants receive active treatment until the children participating in this study reach final adult height.

    About Achondroplasia

    Achondroplasia, the most common form of 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 mutation 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 approved medicines for achondroplasia.

    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 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, the regulatory review of the marketing applications for vosoritide by the U.S. Food and Drug Administration (FDA) and the European Medicines Authority (EMA), the continued clinical development of vosoritide and the timing and conduct of such clinical program. 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 FDA, EMA 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, and future SEC filings and reports by BioMarin. 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-presentation-of-vosoritide-phase-3-data-in-children-with-achondroplasia-at-the-american-society-for-bone-and-mineral-research-2020-annual-meeting-301128169.html

    SOURCE BioMarin Pharmaceutical Inc.

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  4. SAN RAFAEL, Calif., Sept. 8, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia. Achondroplasia is the most common form of disproportionate short stature in humans. The data demonstrated that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles…

    SAN RAFAEL, Calif., Sept. 8, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia. Achondroplasia is the most common form of disproportionate short stature in humans. The data demonstrated that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles. 

    "This study provides the first robust evidence for a precision therapy for achondroplasia that has the potential to fundamentally change the clinical management, growth trajectory, and treatment recommendations for affected children." said Ravi Savarirayan, M.B., B.S., M.D., lead author of The Lancet study and investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria, Australia. "As a treating physician, the lack of therapeutic options for children with achondroplasia represents an unmet medical need in this area."

    The primary endpoint was change from baseline in AGV at 52 weeks in participants administered daily subcutaneous injections of vosoritide, at a dose of 15.0 µg/kg/day, compared with placebo. The findings demonstrated that the adjusted mean difference in AGV between children in the vosoritide group and placebo group was 1.57cm per year in favor of vosoritide (95% CI: 1.22 - 1.93, p value <0·0001), a substantial proportion of the approximately 2 cm/yr AGV deficit relative to average-stature children. The results of subgroup analyses for change from baseline in AGV were consistent with  the overall mean difference between treatment groups in favor of vosoritide, with all 95% CIs overlapping. 

    "The consistency of the 54 months of data from an earlier Phase 2 study and this completed Phase 3 study over 12 months provides a strong data set on the clinical benefits of vosoritide in children with achondroplasia," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "These data are part of a robust clinical program and lay the foundation for a potential first drug therapy to address the underlying molecular pathology in children with achondroplasia. We are looking forward to working with regulatory authorities to address this unmet medical need with a therapeutic choice that demonstrates a meaningful increase in skeletal growth."

    Baseline AGV was calculated from the increase in standing height measured over the last six months of the run-in study. Post-baseline AGV was calculated from standing height at baseline and 52-weeks, and then summarized by treatment arm. As of October 30th, 2019, the 52-week placebo-controlled study was completed, and 119 participants had enrolled in the extension study, where all children are receiving vosoritide.

    Secondary Endpoints, Height Z Score

    A prespecified analysis of the secondary endpoint of change from baseline in height Z score, (which measures the height deficit in standard deviations relative to the mean forage- and gender-matched average stature children), was also performed. The findings demonstrated that the adjusted mean difference in height Z score change from baseline between children in the vosoritide group and placebo group was +0.28 in favor of vosoritide (95% CI: 0.17 – 0.39, p value <0.0001). The results of subgroup analyses were consistent and showed that all estimates of the mean difference between treatment groups were greater than or equal to zero and the 95% CIs  were overlapping.

    There were no adverse effects on, or significant improvements in upper to lower body segment proportionality in children receiving vosoritide during this 52-week study. As expected due to the duration of follow up, no statistically significant changes in secondary endpoints related to wider health outcomes such as quality of life, activities of daily living, and frequency and type of medical and surgical interventions were found. Either a longer treatment period or earlier treatment initiation will be required to detect these changes. As such, an ongoing, open-label, phase 3, extension study will continue to evaluate the balance of benefits and harms of vosoritide until the children reported in this study reach final adult height. This study will collect data regarding vosoritide therapy on wider health measures compared with registry data of untreated children with achondroplasia. This long-term study will also provide data on whether treatment of children with achondroplasia with vosoritide will result in a pubertal growth spurt, which appears to be absent in this condition, and provide the opportunity to detect any harms associated with long-term therapy.

    In addition, a phase 2, randomized, double-blind, placebo-controlled trial of vosoritide in infants and younger children (aged 3 months to <60 months) with achondroplasia has been designed to provide further insights into the long-term treatment effects on body proportionality and growth, as well as how earlier treatment might affect the most substantial medical complications (e.g., foramen magnum stenosis with brainstem compression).

    Safety Summary

    Vosoritide, administered at 15.0 ug/kg/day in this Phase 3 randomized, double-blinded placebo-controlled study over one year, was generally well tolerated. 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. No clinically significant blood pressure decreases, or new safety findings were observed. 

    Regulatory Status

    BioMarin announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application, as well as submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vosoritide. 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 Study

    The global Phase 3 study was a randomized, double-blind, placebo-controlled study of vosoritide in 121 children with achondroplasia aged 5 to 14 for 52 weeks. (The enrollment age criteria were 5 to 18 per the study protocol). Vosoritide is being tested in children whose growth plates are still open. This is approximately 25% of people with achondroplasia. Children in this study have completed a minimum six-month baseline study to determine their baseline growth velocity prior to entering the Phase 3 study. The primary endpoint of the study was the change in growth velocity from baseline over one year in children treated with vosoritide compared to placebo. Children in the study will continue to be evaluated in an ongoing open-label extension study where all study participants receive active treatment until the children participating in this study reach final adult height.

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

    The Phase 2 vosoritide study is a randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia, aged zero to less than 60 months, for a period of 52 weeks. The study will be followed by a subsequent open-label extension trial when all participants receive active treatment. Children in this study will have completed a minimum three-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 Z score. The company also plans to augment the height Z score 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.

    Description of Phase 2 Dose Finding Study

    The primary objectives of the open-label, sequential cohort, dose-finding study were to evaluate the safety and tolerability of daily subcutaneous vosoritide and to determine the dose to carry forward to Phase 3. Secondary objectives were to evaluate the effects of vosoritide on change from pre-treatment baseline in AGV (cm/year), height Z score, and body segment proportionality, the vosoritide pharmacokinetic (PK) profile, and biomarkers of vosoritide activity, and endochondral ossification. All children who completed the 24-month dose finding study were then eligible to continue long term follow up in the ongoing extension study which provides long term evidence of efficacy, durability of effect and safety.

    Lifetime Impact Studies

    BioMarin is conducting lifetime impact of achondroplasia studies in Europe (LIAISE) and Latin America (LISA) to contribute to the understanding of the impact of achondroplasia

    About Achondroplasia

    Achondroplasia, the most common form of 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 mutation 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 approved medicines for achondroplasia.

    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 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 phase 3 study, the consistency of Phase 2 and Phase 3 studies, the requirement of a longer treatment period or earlier treatment initiation to detect changes in secondary endpoints related to wider health outcomes such as quality of life, activities of daily living, and frequency and type of medical and surgical interventions, the expectation that the Phase 2 dose finding study will provide data on whether treatment of children with achondroplasia with vosoritide will result in a pubertal growth spurt and provide the opportunity to detect any harms associated with long term therapy, the continued clinical development of vosoritide and the timing and conduct of such clinical program; the possible results of such studies, and discussions with health authorities about marketing applications. 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: 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, and future SEC filings and reports by BioMarin. 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-the-lancet-publishes-detailed-vosoritide-phase-3-data-demonstrating-statistically-significant-increase-in-annualized-growth-velocity-agv-over-52-weeks-in-children-with-achondroplasia-301125303.html

    SOURCE BioMarin Pharmaceutical Inc.

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  5. SAN RAFAEL, Calif., Sept. 8, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in two 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-two-upcoming-virtual-investor-conferences-301125259.html

    SOURCE BioMarin Pharmaceutical Inc.

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