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 announced the FDA's acceptance of its Biologics License Application (BLA) for the gene therapy valoctocogene roxaparvovec for adults with severe hemophilia A. This therapy, if approved by the PDUFA target date of March 31, 2023, would be the first of its kind in the U.S. BioMarin's application includes extensive clinical data demonstrating effective bleed control. The FDA previously granted Regenerative Medicine Advanced Therapy and Breakthrough Therapy designations for this treatment, highlighting its potential to address unmet medical needs.
BioMarin Pharmaceutical (Nasdaq:BMRN) announced an organizational redesign aimed at enhancing operational efficiency and focusing on R&D investments. This restructuring will lead to a reduction of approximately 120 employees, about 4% of its workforce, resulting in annual cost savings of $50 million from 2023. A one-time cost of $20-$25 million will be incurred due to severance. The company plans to reinvest savings into its product pipeline, including the potential U.S. launch of ROCTAVIAN™ and the continued success of VOXZOGO®.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a conference call and webcast on October 26, 2022, at 4:30 p.m. ET to discuss its third quarter 2022 financial results and provide a general business update. This follows the market's closure on the same day. Interested participants can access the event via BioMarin's investor section on their website. The company focuses on developing innovative therapies for serious genetic diseases, with eight commercial products and numerous candidates in development.
BioMarin Pharmaceutical has resubmitted its Biologics License Application (BLA) for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, to the FDA. This resubmission addresses the FDA's previous Complete Response Letter and includes extensive clinical data, including the global GENEr8-1 Phase 3 study outcomes. If approved, it would become the first gene therapy for this condition in the U.S. The FDA is expected to respond by late October, and the review process could extend beyond the typical six months due to the volume of data provided.
BioMarin has announced that the Institute for Clinical and Economic Review (ICER) reports that its gene therapy Roctavian offers over $4 million in lifetime savings compared to emicizumab for hemophilia A treatment. The therapy, priced at $2.5 million, was evaluated in a model accounting for various treatment costs. BioMarin plans to resubmit its Biologics License Application (BLA) to the FDA by the end of September 2022, with anticipated European sales starting in Q4 2022. The findings from ICER are subject to public comment before a final report is issued.
BioMarin Pharmaceutical announced the European Commission's conditional marketing authorization for ROCTAVIAN™ (valoctocogene roxaparvovec), the first gene therapy for severe hemophilia A. This authorization offers 10 years of market exclusivity based on the therapy's potential significant benefits over existing treatments. ROCTAVIAN is expected to reduce the burden of regular infusions for patients. The approval is supported by extensive clinical data, including results from the GENEr8-1 Phase 3 study, which showed durable bleed control. BioMarin plans a BLA resubmission in the U.S. by the end of September 2022.
BioMarin reported record revenues of $533.8 million for Q2 2022, a 6% increase year-over-year, aided by strong growth from Voxzogo and other franchises. Total revenues for the first half surpassed $1 billion, prompting the company to raise its full-year guidance. Notably, Voxzogo generated $34.4 million in product revenues, while Kuvan revenues declined by 27%. BioMarin anticipates the approval of Roctavian in Europe by Q3 2022 and plans to resubmit its BLA in the U.S. for Roctavian by September.
BioMarin Pharmaceutical (NASDAQ: BMRN) will host a conference call and webcast on August 3, 2022, at 4:30 p.m. ET to discuss its second quarter 2022 financial results and provide a business update. The call can be accessed via U.S. and international dial-in numbers, with a replay available for one week following the event. BioMarin specializes in developing therapies for serious rare diseases, with a portfolio of seven commercial products and multiple candidates in various stages of development.
BioMarin Pharmaceutical presented findings on valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, at the ISTH 2022 World Congress in London. The data support its efficacy and safety over six years, with a potential European Commission approval expected in Q3 2022 following a positive CHMP recommendation. This gene therapy may significantly reduce the burden of disease and treatment for patients. The presentations included comparative effectiveness studies and hemostatic results, showcasing the company's commitment to advancing care for individuals with hemophilia A.
BioMarin Pharmaceutical has received a positive recommendation from the European Medicines Agency's CHMP for its gene therapy, valoctocogene roxaparvovec, targeting severe hemophilia A in adults. This therapy, branded as ROCTAVIAN™, is set to be the first gene therapy approved in Europe for this condition. With over 20,000 adults affected across Europe, the Middle East, and Africa, the company anticipates increased patient access post-approval. The European Commission's final decision is expected in Q3 2022, contingent on the ongoing collection of long-term clinical data.
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