BioMarin's investigational enzyme replacement therapy BMN 401 failed to demonstrate clinical benefit in a Phase 3 trial (ENERGY 3) in children aged 1–12 with ENPP1 deficiency, despite improving a key biomarker (plasma inorganic pyrophosphate). The program now faces substantial regulatory and clinical risk, and analysts warn it may have no clear path to approval. The setback adds to pressure on BioMarin’s rare‑disease portfolio, including recent halts of two Voxzogo trials in other growth‑related conditions due to hip‑injury concerns.
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