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 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.
Entos Pharmaceuticals and BioMarin Pharmaceutical have entered an agreement to use the Fusogenix platform to formulate product candidates for genetic diseases. This collaboration aims to leverage Entos' innovative nucleic acid delivery technology to enhance BioMarin's gene therapy pipeline. Fusogenix employs proprietary lipid formulations for effective delivery of mRNA and DNA. BioMarin will conduct preclinical studies on these formulations to evaluate their therapeutic potential. This partnership underscores BioMarin's commitment to advancing transformative therapies for patients with rare genetic conditions.
BioMarin Pharmaceutical reported third-quarter 2021 revenues of $408.7 million, down 14% from $476.8 million in Q3 2020. Net product revenues fell 12% to $369.5 million, with Kuvan revenues plummeting 45%. GAAP net loss reached $36.5 million, contrasting sharply with a $784.8 million gain in Q3 2020, primarily due to a one-time tax benefit. However, the company anticipates revenue growth in Q4 2021 and a strong 2022, driven by Voxzogo's recent EU approval and upcoming U.S. application. Full-year revenue guidance was revised to $1.820-$1.880 billion.
BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in four virtual investor conferences, as announced on October 25, 2021. Live audio webcasts of the presentations will be accessible via the company's investor relations website. Archived recordings will also be available for a limited time post-conference. BioMarin focuses on developing therapies for rare genetic diseases, with a range of commercial and clinical product candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced a conference call on October 27 at 4:30 p.m. ET to discuss its third quarter 2021 financial results and provide a general business update. The call will be hosted by Jean-Jacques Bienaimé, the company's CEO. Investors can join via a U.S. or international dial-in number or through a live audio webcast on the BioMarin website. A replay will be available for one week after the call.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced the hiring of Harold S. Bernstein as Senior Vice President and Chief Medical Officer, and Ganesh Vedantham as Senior Vice President of Technical Development. Dr. Bernstein, with over 30 years of experience, will lead clinical development and succeed Geoff Nichol. Dr. Vedantham, a leader in CMC functions, will replace Victoria Sluzky, who is retiring after 19 years. CEO Jean-Jacques Bienaimé emphasized their potential to enhance the company’s pipeline for rare genetic diseases.
BioMarin Pharmaceutical announced that the FDA has placed a clinical hold on the BMN 307 Phearless Phase 1/2 study, which evaluates an investigational gene therapy for phenylketonuria (PKU). The hold is due to safety concerns identified in a pre-clinical study, revealing that 6 out of 7 mice given the highest dose developed tumors linked to AAV vector integration. BioMarin will pause further participant enrollment while investigating these findings, although no tumors have been reported in humans. They are committed to monitoring and evaluating the health of participants who received lower doses.
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