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) will present at the BofA Securities 2021 Napa Biopharma Conference on June 16, 2021, at 3:30 PM ET. An audio webcast of the presentation will be available live, with an archived version accessible on the company's website for a limited time afterwards. BioMarin focuses on developing therapies for rare disorders and has a diverse portfolio of six commercialized products alongside several clinical candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical announced its participation in two virtual investor conferences on May 27, 2021. An audio webcast of the presentations will be available live through the company's investor relations website. Additionally, an archived version of the remarks will be accessible for a limited time after the conferences. BioMarin, a global biotechnology company, specializes in innovative therapies for serious rare disorders and has a portfolio of six commercial products alongside multiple clinical candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced that the European Medicines Agency (EMA) has granted accelerated assessment for valoctocogene roxaparvovec, aimed at treating adults with severe hemophilia A. This expedited review process is expected to shorten the evaluation timeline, with a Committee for Medicinal Products for Human Use (CHMP) opinion anticipated in the first half of 2022. BioMarin plans to submit a Marketing Authorization Application (MAA) in June 2021, including 52 weeks of Phase 3 GENEr8-1 trial data.
BioMarin Pharmaceutical has shared a significant update regarding its investigational gene therapy valoctocogene roxaparvovec for severe hemophilia A. The latest data shows sustained treatment benefits over five years, with participants remaining off prophylactic Factor VIII treatment. The mean annualized bleed rate in year five was 0.7, representing a 95% reduction from baseline. The company plans to submit a Marketing Authorization Application in Europe in June 2021 and a Biologics License Application in the U.S. in Q2 2022, contingent on favorable study results.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in two virtual conferences, with live audio webcasts available for investors. The webcasts can be accessed at biomarin.com. An archived version will also be accessible post-conference. BioMarin focuses on developing therapies for rare disorders, boasting a portfolio of six commercial products and various clinical candidates. For more details, visit their website.
BioMarin Pharmaceutical announced three oral and three poster presentations for its investigational gene therapy, valoctocogene roxaparvovec, at the ASGCT Virtual 2021 Annual Meeting. These presentations enhance the understanding of the therapy's potential for treating adults with severe hemophilia A. The company plans to submit a Marketing Authorization Application in Europe and a Biologics License Application in the U.S. by mid-2022, contingent on favorable study results. BioMarin also maintains a strong focus on manufacturing capabilities, aiming to produce 10,000 doses per year.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in two virtual investor conferences on May 3, 2021. Management will present, with an audio webcast available live, and an archived version will be accessible on their website for a limited time post-conference. BioMarin specializes in developing therapies for serious rare disorders, with a portfolio that includes six commercial products and numerous candidates in various stages of development. More information can be found on their official site.
BioMarin Pharmaceutical reported Q1 2021 financial results, showing a 3% decline in total revenues to $486 million compared to Q1 2020. Net product revenues decreased 4% to $417.8 million, with significant drops in Kuvan revenues (-42%) due to generic competition. GAAP net income fell to $17.4 million from $81.4 million, impacted by a prior year asset sale. Positive cash flow of $113.5 million and promising product pipelines, including potential approvals for vosoritide and valoctocogene roxaparvovec, suggest a pivotal year ahead.
BioMarin has partnered with the Allen Institute to develop gene therapies targeting rare genetic diseases of the central nervous system. This collaboration will leverage unique gene delivery technologies, specifically engineered adeno-associated viruses (AAVs) that enhance precision in gene therapy applications. BioMarin will receive exclusive rights for research, development, and commercialization of these therapies. While financial terms were not disclosed, both organizations aim to advance clinical therapy candidates that can significantly impact patient outcomes in the CNS therapeutic area.
BioMarin Pharmaceutical presented data at the 2021 ACMG Annual Clinical Genetics Meeting regarding its investigational treatment, vosoritide, for achondroplasia. The Phase 2 extension study revealed a sustained mean increase in annual growth velocity of 1.35 cm/year over 60 months, with a height Z-score improvement of 0.78. Vosoritide was well tolerated, demonstrating no new adverse events. Additionally, the Lifetime Impact of Achondroplasia in Europe study highlighted increased medical burdens for individuals with achondroplasia, emphasizing the need for ongoing clinical assessments.
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