Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical announced results from a Phase 3 trial of vosoritide, an investigational therapy for achondroplasia, published in The Lancet. Conducted on children aged 5-18, the trial showed that daily subcutaneous vosoritide significantly improved annualized growth velocity (AGV) by 1.57 cm/year compared to placebo (p < 0.0001). Additionally, height Z scores increased by +0.28 (p < 0.0001). The therapy was generally well tolerated with mild adverse effects. BioMarin plans to collaborate with regulatory authorities for potential marketing approvals, addressing a significant unmet medical need.

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.