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. develops and commercializes medicines for rare genetic diseases, with recurring news centered on commercial portfolio performance, regulatory approvals, clinical data and business development. Company updates commonly reference therapies for achondroplasia, phenylketonuria, hemophilia, mucopolysaccharidosis and lysosomal storage diseases, including VOXZOGO, PALYNZIQ, GALAFOLD and POMBILITI + OPFOLDA.
BioMarin news also covers financial results, guidance, acquisition integration, debt financing and governance changes. Clinical and regulatory announcements focus on genetically defined conditions, skeletal disorders, enzyme-based therapies and investigational rare-disease programs such as DMX-200 for focal segmental glomerulosclerosis.
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.
BioMarin Pharmaceutical has received accelerated FDA approval for VOXZOGO™ (vosoritide), aimed at promoting linear growth in children aged five and older with achondroplasia. This approval is based on significant improvements in annualized growth velocity (AGV) observed in clinical trials, where Voxzogo showed an AGV increase of 1.57 cm/year. The therapy, a first-of-its-kind treatment targeting the root cause of achondroplasia, is expected to be available in the US by mid-December. The FDA also issued a Rare Pediatric Disease Priority Review Voucher to BioMarin.