Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical (NASDAQ: BMRN) presented long-term data at ENDO21 showing sustained efficacy of vosoritide in treating achondroplasia. After two years of treatment, children exhibited a mean annual growth velocity (AGV) improvement: 4.28 cm/year at baseline, 5.71 cm/year after one year, and 5.65 cm/year after two years. Retention of participants was high at 93%. Vosoritide, generally well tolerated with mild adverse events, is under review by the FDA and EMA, with a PDUFA action date of August 20, 2021. Additional data may reset this to November 2021.

BioMarin Pharmaceutical announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for adults with severe hemophilia A. This designation is meant to expedite development and review processes for therapies addressing unmet medical needs. The company will provide two years of safety and efficacy data from the Phase 3 GENEr8-1 study to the FDA. RMAT designation complements previous Breakthrough Therapy Designation and allows for more options post-approval based on real-world evidence.

BioMarin Pharmaceutical (NASDAQ:BMRN) has completed enrollment in a global Phase 2 study of vosoritide for treating achondroplasia in children under five. The 52-week, placebo-controlled trial involves about 70 participants and aims to assess safety and growth impact. Vosoritide targets the genetic cause of achondroplasia, addressing a significant treatment gap. The FDA is reviewing a New Drug Application for vosoritide, with a PDUFA date scheduled for August 20, 2021. Positive outcomes could lead to the first pharmacological treatment for this condition.

BioMarin Pharmaceutical (BMRN) reported financial results for Q4 and FY 2020, revealing total revenues of $452.1 million for Q4, nearly flat compared to Q4 2019. Full-year revenues rose 9% to $1.86 billion. Notably, Kuvan revenues fell by 27% due to generic competition, while Palynziq and Naglazyme saw significant growth. GAAP net income was $22.1 million for Q4, with a full-year net income of $859.1 million. The company anticipates 2021 total revenues between $1.75 billion and $1.85 billion, with expected GAAP net loss ranging from $130 million to $80 million. Positive clinical data for valoctocogene roxaparvovec and vosoritide highlight growth potential.

BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in four upcoming virtual investor conferences. Live audio webcasts of the presentations can be accessed via their website, with archived versions available for a limited time post-conference. BioMarin is known for developing therapies for serious rare disorders, boasting a portfolio of six commercial products and several clinical candidates. Investors can find more information about the company and its innovations at www.biomarin.com.

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) has appointed Maykin Ho, Ph.D., a former Goldman Sachs partner, to its Board of Directors. Dr. Ho brings over 30 years of experience in healthcare and finance, aimed at supporting BioMarin's growth in addressing rare genetic diseases. The Chairman and CEO, Jean-Jacques Bienaimé, expressed confidence in Dr. Ho's expertise to enhance the company's strategic direction. BioMarin continues to develop innovative therapies with a portfolio of six commercialized products and numerous candidates in various stages.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced a conference call scheduled for February 25, 2021, at 4:30 p.m. ET. CEO Jean-Jacques Bienaimé will discuss the company's fourth quarter and full year 2020 financial results alongside a business update. The call can be accessed via a live audio webcast on the BioMarin website. For U.S. participants, the dial-in number is (866) 502-9859, while international participants can dial (574) 990-1362, using Conference ID 6488682.

BioMarin Pharmaceutical (BMRN) announced positive topline results from its Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A. The trial included 134 participants and found an 84% reduction in Annualized Bleeding Rate (ABR) after one year, with 80% of participants being bleed-free by week five. Factor VIII infusions also decreased by 99%. The FDA granted Breakthrough Therapy Designation, and BioMarin plans to submit data for marketing approval. The study represents a significant step toward a new treatment paradigm for hemophilia A.