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 reported a 15% increase in total revenues for Q1 2023, reaching $596.4 million, driven by strong sales of VOXZOGO. This product saw revenue growth of 346%, indicating robust market uptake in Japan and Brazil. The company's enzyme product revenues also contributed positively, with ALDURAZYME showing a notable increase.
However, GAAP net income decreased to $50.9 million from $120.8 million in Q1 2022 due to the absence of a previous one-time gain. The company adjusted its revenue guidance for ROCTAVIAN, lowering expectations to between $50 million and $150 million, while reaffirming overall revenue guidance for 2023 between $2.375 billion and $2.5 billion.
BioMarin's operational focus includes preparations for ROCTAVIAN's launch in the U.S., with a PDUFA target action date set for June 30, 2023.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a conference call and webcast on April 26, 2023, at 4:30 p.m. ET to discuss its first-quarter 2023 financial results and provide a business update. CEO Jean-Jacques Bienaimé will lead the session, which can be accessed via the investor section of the BioMarin website. The U.S./Canada dial-in number is 800-831-4163 and the international number is 213-992-4616. Replay options will be available following the call. Founded in 1997, BioMarin focuses on developing therapies for rare genetic disorders, boasting eight commercial products that address unmet medical needs. The company’s innovative approach has produced a diverse pipeline aimed at delivering substantial benefits over existing treatments.
BioMarin Pharmaceutical (BMRN) has announced promising long-term data on VOXZOGO® (vosoritide) for treating achondroplasia. Recent studies show sustained growth velocity improvements over seven years in children treated with VOXZOGO, with annualized growth velocities (AGVs) surpassing untreated peers by 1.90 cm/year for boys and 1.36 cm/year for girls. The drug maintained a favorable safety profile with no new adverse effects. VOXZOGO is the first approved therapy for this condition and is currently under review for younger patients. The latest findings will be presented at the ACMG Annual Meeting in Salt Lake City.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that the FDA has accepted its supplemental New Drug Application (sNDA) to expand the use of VOXZOGO (vosoritide) for treating children under 5 with achondroplasia. This update follows positive results from a global Phase 2 study showing similar safety and efficacy in younger patients compared to older ones. The FDA's PDUFA action date is set for October 21, 2023. Currently, there are no approved pharmacological treatments for this age group in the U.S., potentially expanding access to over 1,000 additional children.
BioMarin Pharmaceutical Inc. announced that the FDA has extended the PDUFA target action date for the Biologics License Application of its gene therapy, ROCTAVIAN (valoctocogene roxaparvovec), for adults with severe hemophilia A to June 30, 2023. This extension follows the submission of a three-year data analysis from the ongoing Phase 3 GENEr8-1 study, which involved 134 participants, marking it as the largest gene therapy trial for hemophilia to date. BioMarin has received multiple designations for this therapy, including Regenerative Medicine Advanced Therapy and Orphan Drug status, reflecting its potential to address significant unmet medical needs.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in three investor conferences in March 2023. Management will be presenting at the Cowen Healthcare Conference on March 8 at 10:30 am ET, followed by the Barclays Global Healthcare Conference on March 15 at 9:30 am ET. Additionally, they will speak at the Jefferies Biotech on the Bay Summit on March 16 in Miami. The events will be webcast live, and replays will be available for a limited time on BioMarin's website.
BioMarin, founded in 1997, focuses on developing therapies for rare genetic disorders, with a strong pipeline of treatments.
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