BioMarin Unveils Promising Initial Data for BMN 401 in ENPP1 Deficiency, Eyes 2027 Launch

Reuters

Sep 08, 2025

BioMarin Unveils Promising Initial Data for BMN 401 in ENPP1 Deficiency, Eyes 2027 Launch

BioMarin Pharmaceutical Inc. has announced new data from multiple studies highlighting its ongoing research in bone health. At the American Society for Bone and Mineral Research 2025 Annual Meeting in Seattle, BioMarin presented results from 14 studies, including those concerning VOXZOGO® (vosoritide). The data demonstrated that treatment with VOXZOGO led to anatomical improvements in spinal morphology in young children with achondroplasia and showed continued efficacy in children who received the medicine after the onset of puberty. Additionally, BioMarin is advancing BMN 401, a potential first-in-disease enzyme therapy for ENPP1 deficiency. The initial pivotal data readout for the ENERGY 3 study in children with ENPP1 deficiency is expected in the first half of 2026, with a potential market launch in 2027.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioMarin Pharmaceutical Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: SF66730) on September 08, 2025, and is solely responsible for the information contained therein.

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