BMRN BioMarin Pharmaceutical Inc.

86.88
-1.11  -1%
Previous Close 87.99
Open 88.56
52 Week Low 68.25
52 Week High 131.945
Market Cap $15,771,297,469
Shares 181,529,667
Float 158,707,851
Enterprise Value $15,926,506,399
Volume 1,145,549
Av. Daily Volume 1,582,794
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
Cerliponase alfa
Batten Disease
Approved
Approved
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
Vosoritide
Achondroplasia
PDUFA
PDUFA
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.

Drug Pipeline

Drug Stage Notes
Valoctocogene roxaparvovec (BMN 270) - GENEr8-1
Hemophilia A
Phase 3
Phase 3
Phase 3 52-week data released January 10, 2021. All endpoints met.
Vosoritide
Children with Achondroplasia at risk of foramen magnum compression
Phase 2
Phase 2
Phase 2 commencement of enrollment announced November 9, 2020.
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., Jan. 10, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced positive topline 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.  This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up. 

    Data from the GENEr8-1 Phase 3 study with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% from a prospectively collected 4.8 (median 2.8) at baseline to 0.8 (median 0.0) bleeding episodes per year (p-value <0.0001), among a pre-specified group of prior participants in a non-interventional baseline observational study (rollover population; N=112). 80% of participants were bleed-free starting at week five after treatment.  

    Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII 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).  

    Table 1:  Mean/Median Annualized Bleeding Rate (ABR) and FVIII Infusion Rate in Phase 3 GENEr8-1 Study Rollover Population (N=112) from Week 5 Through Week 52 at Nov. 2020 Cut Off



    Phase 3

    Rollover Population*

     

    On Factor VIII prophylaxis, before

    valoctocogene roxaparvovec infusion

     

    N=112

    Phase 3

    Rollover Population*

     

    After valoctocogene roxaparvovec

    infusion

     

    N=112



    Mean (SD)

    Median (IQR)

    Mean (SD)

    Median (IQR)

    Annualized

    Bleeding Rate

    (bleeding

    episodes per

    year)

    4.8 (6.5)

    2.8 (0.0, 7.6)

    0.8 (3.0)

    0.0 (0.0, 0.0)

    Annualized

    FVIII  Infusion

    Rate (infusions per

    year)

    135.9 (52.0)

    128.6 (104.1, 159.9)

    2.0 (6.4)

    0.0 (0.0, 0.9)



    *See study descriptions for patient population information.

    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 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. 

    Table 2:  Factor VIII Activity Levels in 6-Month Intervals

    Median Factor

    VIII Activity,

    IU/dL

    Phase 3 Rollover

    Population

    (N=112)

     

    Mean (SD)

    Median

    Phase 3 mITT

    Subset

    Population

    (N=17*)

     

    Mean (SD)

    Median

    Phase 1/2

    6e13 vg/kg

    Cohort

     

    (N=7)

     

    Mean (SD)

    Median

    Phase 1/2

    4e13 vg/kg

    Cohort

     

    (N=6)

     

    Mean (SD)

    Median

    Week 26

    55.1 (57.4)

    38.6

    43.9 (42.1)

    33.8

    71.0 (41.6)

    61.2

    18.0 (8.7)

    18.0

    Week 52

    43.6 (45.3)

    24.2

    42.2 (50.9)

    23.9

    63.6 (36.5)

    60.3

    21.1 (12.3)

    23.8

    Week 76



    27.9 (30.6)

    15.8

    53.9 (31.2)

    50.2

    20.6 (15.4)

    21.3

    Week 104



    24.4 (29.2)

    14.7

    36.4 (26.3)

    26.2

    12.3 (8.2)

    11.6

    *Includes only HIV-negative subjects dosed 2 or more years prior to Nov 2020 data cut date. One participant was lost to follow-up at 66.1 weeks and was henceforth imputed to have a Factor VIII activity of 0 IU/dL through 104 weeks.

    Please see Figure 1: Box-and-Whiskers Plot.

    This is the first statistical evidence demonstrating ABR superiority in a gene therapy trial.  These data give us confidence in this groundbreaking alternative to existing therapies and bring us one step closer to a potential new treatment choice to fulfill an unmet medical need for people with hemophilia A," said Steven W. Pipe, MD, Professor of Pediatrics and Pathology, Coagulation Director, Special Coagulation Laboratory Laurence A. Boxer, MD Research Professor of Pediatrics and Communicable Diseases Department of Pathology Michigan Medicine at the University of Michigan and investigator in the Phase 3 study.  "This data set adds to the growing body of scientific and clinical data around valoctocogene roxaparvovec gene therapy for hemophilia A and creates the possibility for a new treatment paradigm."

    "Over the past seven years, we have conducted rigorous scientific research and clinical programs to address the unmet medical needs of people with severe hemophilia A," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "The decades-long aspirations of the hemophilia community are at the forefront of our ongoing commitment to advance this promising investigational gene therapy for the treatment of severe hemophilia A. We are very encouraged by these data and look forward to working with regulatory authorities, treating physicians, and people with hemophilia A to further understand the potential of this gene therapy."

    "Although factor replacement therapy has been shown to be a safe and effective treatment modality in hemophilia, it suffers both from incomplete prevention of joint disease and having a high treatment burden with recurring needs for intravenous infusions, which can limit important daily activities out of fear of bleeds," said Guy Young, M.D., Director, Hemostasis and Thrombosis Program at Children's Hospital Los Angeles and Professor of Pediatrics (Clinical Scholar), Keck School of Medicine of University of Southern California.  "Novel therapeutic approaches such as gene therapy offer the prospect for both complete prevention of bleeds and subsequent joint damage and eliminating the burden associated with current treatments resulting in an improved quality of life."

    Valoctocogene Roxaparvovec Safety

    Overall, in the Phase 3 study, valoctocogene roxaparvovec has been well tolerated by the 134 participants who received a single 6e13 vg/kg dose. No participants developed inhibitors to Factor VIII, or thromboembolic events.  One participant was lost to follow-up.  Infusion-related reactions were effectively mitigated by managing infusion rates.

    Alanine aminotransferase (ALT) elevation (115 participants, 86%), a laboratory test of liver function, remained the most common adverse event (AE).  Other common adverse events were headache (51 participants, 38%), nausea (50 participants, 37%), aspartate aminotransferase (AST) elevation (47 participants, 35%), arthralgia (38 participants, 28%) and fatigue (37 participants, 27%).  Twenty-two (16.4%) participants experienced a total of 43 serious adverse events (SAEs), and all SAEs resolved. 

    Common, steroid-related side effects can occur with temporary use of corticosteroid (or alternative immunosuppressants) to manage ALT elevation. These side effects have generally been grade 1/2 in intensity, manageable and reversible. Isolated grade 3 steroid-related sides effects (e.g., diabetes, hypertension, weight gain, bone fractures) were observed with longer-term higher dose corticosteroid administration. Corticosteroid-related grade 3 SAEs emerged as a safety issue with extended use of corticosteroids which were reversible with only one event of weight gain ongoing. 

    Overall, in the Phase 1/2 study, the safety profile of valoctocogene roxaparvovec remains consistent with previously reported data with no delayed-onset, treatment-related events.  No participants developed inhibitors to Factor VIII, and no participants withdrew from the study.  No participants have developed thrombotic events.  The most common adverse events associated with valoctocogene roxaparvovec occurred early and included transient infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae. 

    GENEr8-1 Study Description

    The global Phase 3 GENEr8-1 study evaluates superiority of valoctocogene roxaparvovec at the 6e13 vg/kg dose compared to the current standard of care, FVIII prophylactic therapy.  All study participants had severe hemophilia A at baseline, defined as less than or equal to 1 IU/dL of Factor VIII activity.  The study included 134 total participants, all of whom had a minimum of 12 months of follow-up at the time of the datacut.  The first 22 participants were directly enrolled into the Phase 3 study, 17 of whom were HIV-negative and dosed at least 2 years prior to the datacut date (referred to as the subset).  The remaining 112 participants (rollover population) completed at least six months in a separate non-interventional study to prospectively assess bleeding episodes, Factor VIII use, and health-related quality of life while receiving Factor VIII prophylaxis prior to rolling over to receive a single infusion of valoctocogene roxaparvovec in the GENEr8-1 study.

    Regulatory Status

    BioMarin is working with the U.S. Food and Drug Administration (FDA) to align on steps forward to obtain marketing approval for valoctocogene roxaparvovec gene therapy for severe hemophilia A. 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. Additionally, the European Medicines Agency (EMA) requested one-year results from the full Phase 3 study to inform their benefit-risk assessment. To facilitate this submission within the EMA regulatory framework, BioMarin withdrew the MAA and plans to resubmit the MAA with these data to the EMA in the second quarter of 2021 following discussions with the Agency.

    The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy Designation.  BioMarin's valoctocogene roxaparvovec 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. 

    Call and Webinar to be Held Today, January 10, 2021 at 7:15 PM Eastern Time

    Join from a PC, Mac, iPad, iPhone or Android device:

    Please click here to join a live Zoom video webinar at 7:15 PM Eastern.

    Or join by phone:

    Dial (for higher quality, dial a number based on your current location):

    US: +1 669 900 6833  or +1 346 248 7799  or +1 253 215 8782  or +1 312 626 6799  or +1 929 205 6099  or +1 301 715 8592

    Zoom Webinar ID: 959 1943 2167

    International numbers available here.  

    Phase 1/2 Dose Escalation Study Description

    The Phase 1/2 dose escalation study is ongoing and continues to monitor participants long-term.  In the study, a total of 15 patients with severe hemophilia A and Factor VIII activity levels less than or equal to 1 IU/dL received a single dose of BMN 270, seven of whom were treated at a dose of 6e13 vg/kg and six of whom were treated at a lower dose of 4e13 vg/kg.  The other two participants were treated at lower doses as part of dose escalation in the study and did not achieve therapeutic efficacy.  

    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, and the Phase 3 results particularly; the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, the potential for valoctocogene roxaparvovec to reduce or eliminate bleeds, reduce the number of Factor VIII infusions, and the ongoing clinical programs generally.  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 the above data and additional 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 BioMarin's Annual and quarterly Reports on Forms 10-K and 10-Q, and future 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-positive-phase-3-gene-therapy-trial-results-in-adults-with-severe-hemophilia-a-study-met-all-primary-and-secondary-efficacy-endpoints-in-one-year-data-set-301204797.html

    SOURCE BioMarin Pharmaceutical Inc.

    View Full Article Hide Full Article
  2. SAN RAFAEL, Calif., Jan. 5, 2021 /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-301200463.html

    SOURCE BioMarin Pharmaceutical Inc.

    View Full Article Hide Full Article
  3. SAN RAFAEL, Calif., Dec. 21, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today 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.  These analyses are the result of the combination of data of the same patients enrolled in three consecutive trials.  In the first trial, a "run in" period consisted of longitudinal measurement of height in all patients prior to receiving treatment.  After at least six months observation in the run-in trial, 121 patients were randomized 1:1 to receive either placebo…

    SAN RAFAEL, Calif., Dec. 21, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today 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.  These analyses are the result of the combination of data of the same patients enrolled in three consecutive trials.  In the first trial, a "run in" period consisted of longitudinal measurement of height in all patients prior to receiving treatment.  After at least six months observation in the run-in trial, 121 patients were randomized 1:1 to receive either placebo or vosoritide at a dose of 15 ug/kg/day.  One year later, patients previously receiving placebo were crossed over to receive vosoritide in an open-label treatment extension study, while those patients previously on vosoritide remained on treatment. 

    A first 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 compared to untreated children, which is the sum of the first year (1.73) and the second year (1.79). 

    Yearly change in standing height (cm)

    Year 1

    Year 2

    Untreated (N=38*), mean (SD)

    3.96 (0.92)

    3.82 (0.99)

    Vosoritide (N=52**), mean (SD)

    5.69 (0.97)

    5.61 (1.09)

    Treatment effect (95% CI)

    1.73 (1.33 - 2.14)

    1.79 (1.35 - 2.24)

    P-value***

    <0.0001

    <0.0001



    *38 participants were enrolled in the run-in study more than 12 months and were at least 5 years of age at that point of time in advance of randomization and therefore contribute at least two years of evaluation of height in the absence of treatment.

     

     **Data from 6 patients were unavailable due to patient withdrawals during Year 2 (n=2) and due to restrictions in study conduct because of Covid-19 (n=4).  The patients whose data are not available due to Covid-19 are still on treatment.

     

    *** p-value for unadjusted treatment effect.

    A second supportive analysis evaluated the treatment effect of vosoritide administered continuously for over two years, including all children regardless of the duration of prior observation (N=119; 58 treated and 61 untreated children). This analysis showed a mean improvement in AGV  in the vosoritide treated group of 1.69 cm/year, compared with untreated subjects, calculated over the entire observation period.  A similar method was used in the analysis of the effect of one year's treatment with vosoritide previously published in The Lancet on Sept. 5, 2020, demonstrating a placebo-adjusted improvement in AGV of 1.57 cm/year.  (Table 2 in Lancet publication, DOI:  https://doi.org/10.1016/S0140-6736(20)31541-5 ). 

    In the vosoritide treated group, AGV declined by -0.14 cm/year during the second year of vosoritide treatment compared to the baseline AGV established in the 6 months prior to the first year of treatment. This decline is similar to the annual AGV decline with age that has been observed in natural history studies, as well as during one year of treatment with placebo (-0.12 cm/year), further supporting the maintenance of treatment effect. 

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

    Vosoritide was generally well tolerated with no new safety concerns.  Serious adverse events observed in the trial were representative of common childhood illnesses and were deemed unrelated to vosoritide.  No new safety findings have emerged, and clinically inconsequential blood pressure changes were mild, transient and self-limiting.

    "BioMarin is committed to the long-term follow up of children participating in vosoritide studies and the overall health of people with achondroplasia.  We look forward to sharing more data on wider health measures that either require a longer treatment period or starting treatment at a younger age.  We are also specifically studying the impact of vosoritide in infants at risk of serious and potentially fatal medical complications related to achondroplasia," said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin.  "We are grateful for the support of the children and their families who are in these studies, the clinical trial investigators and their staffs, BioMarin employees, and the community.  We look forward to sharing more detailed information at an upcoming medical meeting and further contributing to the scientific body of knowledge about vosoritide and its potential impact over time."

    "Follow up data from extension studies are critical to expanding our understanding of the wider impact 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.  "BioMarin has developed a comprehensive clinical program designed to address the effects on health and day to day living by evaluating proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension."

    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.   No clinically significant blood pressure decreases or new safety findings were observed. 

    Regulatory Status

    BioMarin's marketing applications for vosoritide are currently under review by both the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and if approved would be the first therapy for achondroplasia in the U.S. and Europe.  The FDA's Prescription Drug User Fee Act target action date is August 20, 2021.  The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in the second half of 2021.

    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.

    Robust Clinical Program

    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 pivotal phase 3 study and 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.

    Description of Phase 2 Study for Children at Risk of Life-Threatening Foramen Magnum Compression

    This is a Phase 2 randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of vosoritide in infants with achondroplasia at risk of requiring cervicomedullary decompression surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. In addition, the study will also measure a secondary endpoint to evaluate the effect of vosoritide on growth of the foramen magnum volume through MRI scans.  Within a month of study initiation in November, two of the planned 20 (10%) participants enrolled. 

    Foramen magnum compression is the foremost life-threatening complication of achondroplasia in infants. This study investigates the safety of vosoritide in infants aged 0 -1 years of age with achondroplasia who have evidence of foramen magnum compression at-risk for requiring cervicomedullary decompression surgery. Those infants are under close observation for the appearance of neurological signs of progressive foramen magnum compression, and the current standard of care is palliative with many eventually requiring surgery. The study aims to enroll approximately 20 infants, who will be randomized to either current standard of care plus vosoritide treatment or current standard of care alone for a two-year period.  After the two-year randomized period, children in the study would be eligible to receive vosoritide in an open-label, 3-year additional extension period. The study will examine the incidence of adverse events between the two groups, volume MRI measurements of the foramen magnum, skull and spine, and progression to requiring decompression surgery.

    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 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 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 Z-scores, which is the number of standard deviations in relation to the mean height of age- and gender-matched, average stature children.  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.  Currently, cohorts 1 and 2 are fully enrolled and cohort 3 is 85% (17/20)  enrolled.  The remaining study participants to enroll are in the observational period and are expected to be dosed in 1Q 2021.

    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 maintenance of AGV after two years, the similarity of AGV and height gain in the first and second years of the Phase 3 study, the similarity of AGV and height gain to earlier studies, the continued clinical development of vosoritide and the timing and conduct of such clinical program; the enrollment expectations for ongoing clinical trials; the possible results of such studies, the timing of decisions by health authorities about marketing applications, and the Company's plans to discuss provision of the two-year data with health authorities. 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 Quarterly Report on Form 10-Q for the quarter ended September 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-benefit-maintained-for-over-two-years-in-children-with-achondroplasia-treated-with-vosoritide-in-phase-3-extension-study-301196598.html

    SOURCE BioMarin Pharmaceutical Inc.

    View Full Article Hide Full Article
  4. NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Nasdaq (NASDAQ:NDAQ) today announced the results of the annual reconstitution of the Nasdaq-100 Index® (NASDAQ:NDX), which will become effective prior to market open on Monday, December 21, 2020.

    The following six companies will be added to the Index: American Electric Power Company, Inc. (NASDAQ:AEP), Marvell Technology Group Ltd. (NASDAQ:MRVL), Match Group, Inc. (NASDAQ:MTCH), Okta, Inc. (NASDAQ:OKTA), Peloton Interactive, Inc. (NASDAQ:PTON), Atlassian Corporation Plc (NASDAQ:TEAM).

    The Nasdaq-100 Index is composed of the 100 largest non-financial companies listed on The Nasdaq Stock Market® and dates to January 1985 when it was launched along with the Nasdaq Financial-100 Index®, which is comprised…

    NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Nasdaq (NASDAQ:NDAQ) today announced the results of the annual reconstitution of the Nasdaq-100 Index® (NASDAQ:NDX), which will become effective prior to market open on Monday, December 21, 2020.

    The following six companies will be added to the Index: American Electric Power Company, Inc. (NASDAQ:AEP), Marvell Technology Group Ltd. (NASDAQ:MRVL), Match Group, Inc. (NASDAQ:MTCH), Okta, Inc. (NASDAQ:OKTA), Peloton Interactive, Inc. (NASDAQ:PTON), Atlassian Corporation Plc (NASDAQ:TEAM).

    The Nasdaq-100 Index is composed of the 100 largest non-financial companies listed on The Nasdaq Stock Market® and dates to January 1985 when it was launched along with the Nasdaq Financial-100 Index®, which is comprised of the 100 largest financial stocks on Nasdaq®. These indexes act as benchmarks for financial products such as options, futures, and funds. The Nasdaq-100 is reconstituted each year in December, timed to coincide with the quadruple witch expiration Friday of the quarter.

    The Nasdaq-100 Index is the basis of the Invesco QQQ Trust (NASDAQ:QQQ) which aims to provide investment results that, before expenses, correspond with the Nasdaq-100 Index performance. In addition, options, futures and structured products based on the Nasdaq-100 Index and the Invesco QQQ Trust trade on various exchanges.

    As a result of the reconstitution, the following six companies will be removed from the Index: BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), Citrix Systems, Inc. (NASDAQ:CTXS), Expedia Group, Inc. (NASDAQ:EXPE), Liberty Global plc (NASDAQ:LBTYA), Take-Two Interactive Software, Inc. (NASDAQ:TTWO), Ulta Beauty, Inc. (NASDAQ:ULTA).

    Information

    For information about the six companies to be added to the Nasdaq-100 Index, please visit the following respective company websites:

    American Electric Power Company, Inc. – https://www.aep.com/

    Marvell Technology Group Ltd. – https://www.marvell.com/

    Match Group, Inc. – https://www.mtch.com/

    Okta, Inc. – https://www.okta.com/

    Peloton Interactive, Inc. – https://www.onepeloton.com/

    Atlassian Corporation Plc – https://www.atlassian.com/

    About Nasdaq Global Indexes

    Nasdaq Global Indexes has been creating innovative, market-leading, transparent indexes since 1971. Today, our index offering spans geographies and asset classes and includes diverse families such as the Dividend and Income (includes Dividend Achievers), Dorsey Wright, Fixed Income (includes BulletShares®), Global Equity, Green Economy, Nordic and Commodity indexes. We continuously offer new opportunities for financial product sponsors across a wide-spectrum of investable products and for asset managers to measure risk and performance. Nasdaq also provides exchange listing, custom index and design solutions to financial organizations worldwide.

    About Nasdaq

    Nasdaq (NASDAQ:NDAQ) is a global technology company serving the capital markets and other industries. Our diverse offering of data, analytics, software and services enables clients to optimize and execute their business vision with confidence. To learn more about the company, technology solutions and career opportunities, visit us on LinkedIn, on Twitter @Nasdaq, or at www.nasdaq.com.

    Media Relations Contact

    Emily Pan

    (646) 637-3964

    Issuer & Investor Contact

    Index Client Services

    The information contained above is provided for informational and educational purposes only, and nothing contained herein should be construed as investment advice, either on behalf of a particular financial product or an overall investment strategy. Neither Nasdaq, Inc. nor any of its affiliates makes any recommendation to buy or sell any financial product or any representation about the financial condition of any company or fund. Statements regarding Nasdaq's proprietary indexes are not guarantees of future performance. Actual results may differ materially from those expressed or implied. Past performance is not indicative of future results. Investors should undertake their own due diligence and carefully evaluate companies before investing. ADVICE FROM A SECURITIES PROFESSIONAL IS STRONGLY ADVISED.

    -NDAQG-



    Primary Logo

    View Full Article Hide Full Article
  5. SAN RAFAEL, Calif., Dec. 1, 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-301182457.html

    SOURCE BioMarin Pharmaceutical Inc.

    View Full Article Hide Full Article
View All BioMarin Pharmaceutical Inc. News