On September 15, 2026, BioMarin Pharmaceutical Inc. announced the discontinuation of development for VOXZOGO in children with Noonan syndrome. The decision followed an assessment of study feasibility and the current treatment landscape, rather than safety or efficacy concerns regarding the drug. This strategic withdrawal impacts a specific pediatric indication but does not alter the company's broader pipeline or commercial operations. BioMarin Pharmaceutical Inc. operates in the biotechnology industry, focusing on the discovery, development, and commercialization of therapies for rare genetic diseases.

On September 10, 2026, BioMarin Pharmaceutical Inc.'s Board of Directors increased its size from ten to eleven members and appointed Robert Plenge, M.D., Ph.D., as an independent non-employee director. Dr. Plenge was simultaneously appointed to the Science and Technology Committee. In connection with his appointment, he received restricted stock units valued at $400,000 under the Company’s 2017 Equity Incentive Plan, vesting prior to the next annual meeting. He will receive standard director fees and committee retainers as outlined in the company's proxy statement. BioMarin Pharmaceutical Inc. operates in the biotechnology industry, focusing on the discovery, development, and commercialization of therapies for rare genetic diseases.

BioMarin Pharmaceutical Inc. completed its acquisition of Alesta Therapeutics B.V. on August 31, 2026, making Alesta a wholly owned subsidiary. The transaction was executed pursuant to a Share Purchase Agreement previously announced on August 18, 2026. BioMarin operates in the biotechnology industry, focusing on the development and commercialization of therapies for rare diseases.

BioMarin entered into a binding term sheet with Ascendis Pharma to resolve all pending global patent litigation regarding the TransCon CNP asset, navepegritide (Yuviwel). Under the agreement, BioMarin grants Ascendis a non-exclusive, worldwide license for Yuviwel across all indications. In exchange, Ascendis will pay royalties of 20% on U.S. net sales and 18% on net sales in the EU, Brazil, and South Korea until May 2030. The settlement includes retroactive royalty application from first commercial sale and dismissal of all related legal proceedings. BioMarin is a biotechnology company focused on developing treatments for rare genetic diseases.

BioMarin Pharmaceutical Inc. entered into a Share Purchase Agreement on August 17, 2026, to acquire Alesta Therapeutics B.V. for an upfront cash payment of $275 million and up to $215 million in future development and regulatory milestones. The transaction is expected to close in the third quarter of 2026 and will provide BioMarin with ALE1, a clinical-stage oral small molecule therapy for hypophosphatasia currently in Phase 1/2a trials. Alesta will spin out all non-ALE1 assets to a new entity prior to closing, meaning no Alesta employees will transfer to BioMarin. The company intends to fund the acquisition with cash on hand and expects to provide updated full-year 2026 guidance after the close. BioMarin Pharmaceutical Inc. is a global rare disease biotechnology company focused on developing medicines for genetically defined conditions.

BioMarin Pharmaceutical Inc. reported second quarter 2026 financial results on August 6, 2026, following its April 27 acquisition of Amicus Therapeutics. Total revenues increased 20% year-over-year to $990 million, driven by new products GALAFOLD and POMBILITI + OPFOLDA alongside strong VOXZOGO demand. GAAP net income decreased 81% to $45 million due to acquisition-related costs, intangible asset amortization, and interest expense from transaction financing. The company raised its full-year 2026 total revenue guidance midpoint to $3,900 million and increased VOXZogo revenue expectations to at least $1 billion. BioMarin is a rare disease biotechnology company focused on developing and commercializing innovative medicines for genetically defined conditions.