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 participate in two virtual investor conferences, as announced on September 8, 2020. An audio webcast of the presentations will be available live, with access through their investor website. Archived versions of the remarks will also be provided for a limited time post-conference. BioMarin is a global biotechnology company focused on rare disorders, with six commercial products and several candidates in clinical development.
BioMarin Pharmaceutical (BMRN) announced the submission of a New Drug Application (NDA) to the FDA for vosoritide, a potential treatment for children with achondroplasia. This regulatory move follows the validation of the Marketing Authorization Application (MAA) by the EMA on Aug. 13, 2020. Vosoritide represents a significant milestone as the first pharmacological treatment option for this condition, which affects children characterized by disproportionate short stature. The drug is currently being tested in children under 18 with open growth plates, comprising 25% of achondroplasia cases.
BioMarin Pharmaceutical announced that the FDA issued a Complete Response Letter (CRL) regarding its Biologics License Application for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020. The FDA's CRL indicated that additional data, specifically two years from the ongoing Phase 3 study, is required to demonstrate the therapy's durability of effect. Although BioMarin plans to meet with the FDA to discuss next steps, the company's previous expectations were altered with this new requirement. The European Medicines Agency's review of the therapy is still ongoing.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced validation from the European Medicines Agency (EMA) for its Marketing Authorization Application (MAA) for vosoritide, a treatment aimed at children with achondroplasia, a common form of disproportionate short stature. The MAA review began on August 13, 2020. The company is also set to submit a New Drug Application (NDA) to the FDA in the third quarter of 2020. Vosoritide offers potential as the first pharmacological intervention for achondroplasia, which affects one in 25,000 live births globally.
BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in two virtual investor conferences: the Wedbush PacGrow Healthcare Virtual Conference on August 11, 2020, at 10:55 AM ET and the Canaccord Genuity 40th Annual Growth Conference on August 13, 2020, at 1:00 PM ET. Key executives, including Jeff Ajer and Brian Mueller, will represent the company. Live audio webcasts will be accessible on their investor relations website, with archived versions available afterwards. BioMarin focuses on innovative therapies for serious rare disorders.
BioMarin Pharmaceutical (BMRN) reported its Q2 2020 financial results, with total revenues rising to $429.5 million, an 11% increase year-over-year. Net product revenues grew by 4% to $386.8 million, driven by significant increases in Palynziq (116%) and Aldurazyme (457%). However, Vimizim and Naglazyme revenues fell by 5% and 18%, respectively. The company reported a GAAP net loss of $29.2 million, an improvement from a loss of $37.4 million last year. With cash reserves reaching $1.7 billion, BioMarin is focused on upcoming product approvals, particularly for its gene therapies. Full-year GAAP net income guidance is now set at $720-$980 million.
BioMarin Pharmaceutical (NASDAQ: BMRN) submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vosoritide, a treatment aimed at children with achondroplasia. This application follows a Phase 3 study confirming its safety and efficacy. If approved, vosoritide will be the first medicine targeting achondroplasia in Europe. The company plans to file a New Drug Application (NDA) with the FDA in Q3 2020. Vosoritide has received Orphan Drug designation from the FDA and EMA, highlighting its significance in addressing a rare condition.
BioMarin Pharmaceutical will host a conference call on August 4 at 4:30 p.m. ET to discuss its second quarter 2020 financial results and provide a business update. The call will feature Jean-Jacques Bienaimé, CEO, and will be available for replay on the company's website for one week after the event. Interested parties can join via the provided U.S. and international dial-in numbers.
BioMarin Pharmaceutical has promoted Brian R. Mueller to Executive Vice President, Chief Financial Officer, and Andrea L. Acosta to Group Vice President, Chief Accounting Officer. Mueller has been with the company for 17 years and served as acting CFO since February 2020. The CEO, Jean-Jacques Bienaimé, expressed confidence in Mueller's leadership and the depth of talent within the finance organization. Mueller aims to support the development of therapies for rare diseases, while Acosta emphasizes the need for a finance team sensitive to innovation requirements.
BioMarin Pharmaceutical announced participation in the BofA Securities 2020 Napa Biopharma Conference on June 24, 2020, at 11:30 AM ET. Key representatives, Henry Fuchs, M.D., President of Worldwide Research & Development, and Brian Mueller, Acting CFO, will present. The event will feature a live audio webcast accessible via the company's investor site. BioMarin focuses on innovative therapies for serious rare disorders, with six commercialized products and numerous candidates in development.
FAQ
What is the current stock price of BioMarin Pharmaceuticals (BMRN)?
What is the market cap of BioMarin Pharmaceuticals (BMRN)?
What is BioMarin Pharmaceuticals Inc. known for?
Which products are marketed by BioMarin independently?
What recent product approvals has BioMarin received?
How does BioMarin support patients with achondroplasia?
What are BioMarin's key strategic initiatives for growth?
How is BioMarin addressing hypochondroplasia?
What financial growth has BioMarin reported recently?
What collaborations does BioMarin have for product marketing?
How can patients get support for BioMarin's products?
Where can I find more information about BioMarin?
