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BIOMARIN PHARMACEUTICAL INC.

(BMRN)
  Report
Real-time Estimate Cboe BZX  -  01:09:06 2023-02-07 pm EST
111.02 USD   -1.03%
08:01aBiomarin To Host Fourth Quarter And Full-year 2022 Financial Results Conference Call And Webcast On Monday, February 27, At 4 : 30 p.m. ET
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02/03SVB Securities Adjusts BioMarin Pharmaceutical's Price Target to $130 From $122, Maintains Outperform Rating
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01/30BMO Capital Initiates BioMarin Pharmaceutical at Market Perform With $107 Price Target
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BioMarin Pharmaceutical Inc. Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

11/23/2022 | 08:29am EST

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration (FDA) review of the Biologics License Application (BLA) of ROCTAVIAN™ (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. 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 is also conducting a Phase 3, 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 severe hemophilia A (Study 270-303).

Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors (Study 270-205). 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 (Factor VIII 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 (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 injections or shots 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.


ę S&P Capital IQ 2022
All news about BIOMARIN PHARMACEUTICAL INC.
08:01aBiomarin To Host Fourth Quarter And : 30 p.m. ET
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02/03SVB Securities Adjusts BioMarin Pharmaceutical's Price Target to $130 From $122, Mainta..
MT
01/30BMO Capital Initiates BioMarin Pharmaceutical at Market Perform With $107 Price Target
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01/24Morgan Stanley Raises Price Target on BioMarin Pharmaceutical to $132 From $113, Mainta..
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01/19Insider Sell: Biomarin Pharmaceutical
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01/09BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN in Largest..
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01/08BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN™ in ..
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01/08Biomarin Pharmaceutical Inc. Announces Stable and Durable Annualized Bleed Control for ..
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Financials (USD)
Sales 2022 2 095 M - -
Net income 2022 130 M - -
Net cash 2022 304 M - -
P/E ratio 2022 165x
Yield 2022 -
Capitalization 20 849 M 20 849 M -
EV / Sales 2022 9,81x
EV / Sales 2023 7,83x
Nbr of Employees 3 045
Free-Float 99,3%
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Number of Analysts 24
Last Close Price 112,18 $
Average target price 122,30 $
Spread / Average Target 9,03%
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Managers and Directors
Jean-Jacques BienaimÚ Chairman & Chief Executive Officer
Brian R. Mueller Chief Financial Officer & Executive VP-Finance
Henry J. Fuchs President-Worldwide Research & Development
Eduardo E. von Pervieux Chief Information Officer
C. Greg Guyer Chief Technical Officer & Executive Vice President
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