Welcome to our dedicated page for Biomarin Pharmaceutical news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on Biomarin Pharmaceutical stock.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a global rare disease biotechnology company focused on medicines for genetically defined conditions. This news page aggregates company announcements, press releases and event updates so readers can follow how BioMarin’s strategy, pipeline and commercial portfolio evolve over time.
Recent communications from BioMarin highlight its emphasis on Enzyme Therapies and Skeletal Conditions business units, as well as its work with ROCTAVIAN, a gene therapy for hemophilia A. The company issues news on topics such as financial results, long-term guidance, corporate strategy, business development transactions, clinical data presentations and regulatory milestones for its therapies.
Examples of news themes include updates on VOXZOGO in achondroplasia and other skeletal conditions, progress with PALYNZIQ in phenylketonuria, development of BMN 401 for ENPP1 deficiency and other pipeline programs in rare genetic diseases. BioMarin also reports on strategic partnerships, such as digital and data collaborations, and on planned acquisitions intended to expand and diversify its rare disease portfolio.
Investors and followers of BMRN can use this page to review earnings announcements, conference presentations, investor day materials and product-specific updates released through newswires. By checking this feed regularly, readers can see how BioMarin describes its growth priorities, clinical milestones and capital allocation decisions across its rare disease franchises.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a conference call and webcast on February 23, 2022, at 4:30 p.m. ET to discuss its fourth quarter and full year 2021 financial results. The event will feature insights from CEO Jean-Jacques Bienaimé, covering key financial metrics and a general business update. Investors can access the live audio via the company’s investor section at www.biomarin.com. A replay will be available for one week following the call.
BioMarin Pharmaceutical has announced the sale of its Rare Pediatric Disease Priority Review Voucher for $110 million to an undisclosed purchaser. The voucher was obtained in November 2021 through the FDA program aimed at promoting treatments for rare pediatric diseases, awarded following the approval of VOXZOGO™ for treating achondroplasia. BioMarin plans to reinvest the proceeds into its pipeline of therapies and expects the sale to contribute positively to its GAAP net income in 2022.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced positive outcomes from a two-year analysis of the Phase 3 GENEr8-1 study at the EAHAD Congress. The study, which involved 134 participants, showcased a significant reduction in the Annualized Bleeding Rate (ABR) by 4.1 bleeds per year (85% decrease) and a 98% reduction in the Factor VIII infusion rate. Notably, 95% of participants remained off prophylactic therapy. No major safety concerns were reported, with most adverse events being transient. These findings highlight the potential of valoctocogene roxaparvovec as a transformative treatment for severe hemophilia A.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented data on valoctocogene roxaparvovec, an investigational gene therapy for severe hemophilia A, at the 15th Annual Virtual Congress of EAHAD from February 2-4, 2022. Key highlights include an oral presentation of a two-year analysis from the Phase 3 GENEr8-1 study, which involved 134 participants, showcasing significant safety data. Additionally, three posters related to health-related quality of life, gene transfer in participants with HIV, and patient preferences in hemophilia therapy were also shared. The presentations aim to enhance understanding of this novel treatment.
BioMarin Pharmaceutical announced promising results from its Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A. With 134 participants, the study demonstrated an 85% reduction in Annualized Bleeding Rate (ABR) and a significant decrease in Factor VIII infusion rates. At two years, the mean endogenous Factor VIII activity was reported at 23.0 IU/dL. No new safety concerns emerged, and the therapy was well tolerated. BioMarin plans to submit two-year follow-up safety and efficacy data to the FDA and anticipates further discussions regarding the therapy's approval.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced that its CEO, Jean-Jacques Bienaimé, will present at two virtual investor conferences in January 2022. The webcasts of these events will be accessible through their investor relations webpage, with archived versions available for a limited time post-conference. BioMarin focuses on developing therapies for serious rare disorders, currently offering seven commercialized products and several candidates in various stages of clinical development. For more information, visit their official website.
BioMarin Pharmaceutical and Skyline Therapeutics have entered a multi-year collaboration to develop Adeno-Associated Virus (AAV) gene therapies targeting genetic cardiovascular diseases, specifically dilated cardiomyopathies (DCM). BioMarin will leverage its gene therapy expertise and Skyline's vector engineering capabilities. Skyline will receive an undisclosed upfront payment, equity investment, and milestone payments. BioMarin retains commercialization rights in the U.S., Europe, and Latin America, while Skyline will oversee the Asia-Pacific region. This partnership aims to address significant unmet medical needs in DCM.
BioMarin Pharmaceutical announced the appointment of Mark Alles, former CEO of Celgene, to its Board of Directors from January 1, 2022. This strategic addition aims to enhance the board's expertise in operational, financial, and business development domains. Jean-Jacques Bienaimé, the current CEO, expressed enthusiasm for Mark's contributions to advancing innovative therapies for rare diseases. Alles brings extensive industry experience, having held prominent roles at Celgene and other pharmaceutical companies.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a virtual R&D Day on November 30, 2021, at 8:00 am PT. This event aims to inform the investment community about the company's earlier-stage development portfolio, focusing on innovative therapies for genetic diseases. External experts will join BioMarin's management to share insights on translating genetic discoveries into transformative medicines. The presentation will be accessible via YouTube, with a replay available on BioMarin's investor relations website.
BioMarin Pharmaceutical (NASDAQ: BMRN) announces participation in two upcoming virtual investor conferences, scheduled for December 2021. An audio webcast will be available live, and archived presentations can be accessed on their website for a limited time. BioMarin specializes in innovative therapies for rare genetic diseases, boasting a diverse portfolio of commercial and clinical products. For more details, visit their website.