Welcome to our dedicated page for BioMarin Pharmaceuticals news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on BioMarin Pharmaceuticals stock.
BioMarin Pharmaceuticals Inc. (symbol: BMRN) is a global biotechnology firm dedicated to the development and commercialization of first-in-class and best-in-class therapeutics. Founded in 1997, the company's mission is to transform the lives of patients suffering from serious and life-threatening rare genetic diseases, particularly those affecting children. BioMarin targets rare conditions that are often inherited, difficult to diagnose, progressively debilitating, and lack effective treatment options.
BioMarin's diverse product portfolio includes independently marketed therapies such as Naglazyme, Vimizim, and Brineura, as well as Kuvan and Palynziq for treating phenylketonuria (PKU). The company also collaborates with Genzyme to market Aldurazyme. In 2021, BioMarin's Voxzogo (vosoritide) was approved for the treatment of achondroplasia, a rare genetic disorder affecting bone growth. Following this, in 2022, Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for hemophilia A, received approval in Europe and in the U.S. in 2023.
BioMarin places significant emphasis on research, development, manufacturing, and commercialization speed, aiming to deliver important therapies promptly. The company's capabilities have led to numerous transformational therapies addressing unmet medical needs, providing substantial benefits over existing treatments.
Notable recent developments include the expanded FDA approval for Voxzogo to increase linear growth in pediatric patients of all ages with achondroplasia with open epiphyses, and the substantial revenue growth contributed by global demand for Voxzogo. BioMarin also focuses on hypochondroplasia, launching a pivotal clinical trial program and presenting positive early results from a Phase 2 study.
Financially, BioMarin has demonstrated robust growth, with increased revenue driven by high demand for its products. The company has secured agreements with U.S. and European health authorities to expand patient access to Roctavian and other treatments. Strategic initiatives include corporate governance enhancements and collaborations with major investors to maximize long-term value creation.
For further details about ongoing clinical trials, product information, and patient support, visit www.biomarin.com.
BioMarin Pharmaceutical (NASDAQ: BMRN) provided updates on its gene therapy programs on February 17, 2022. The FDA has requested additional data to lift the clinical hold on the BMN 307 Phase 1/2 study for phenylketonuria, which may take several quarters to assess. Meanwhile, enrollment is complete for the Phase 3 study of valoctocogene roxaparvovec for hemophilia A, with results expected in 1H 2023. The company continues to advance multiple trials, including for BMN 331, which has received Orphan Disease Designation.
BioMarin Pharmaceutical announced participation in two virtual investor conferences on February 16, 2022. The management will present, and a live audio webcast of the presentations can be accessed at the official website. An archived version will also be available after the events. BioMarin, focusing on innovative therapies for serious rare disorders, has a diverse portfolio with seven commercial products. For more details, visit www.biomarin.com.
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.
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