BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

Reynald Castaneda / endpoints - BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don't produce enough of the ENPP1 enzyme, which generates plasma inorganic pyrophosphate .…

AI Summary: BioMarin reported a Phase 3 trial that failed to show clinical benefit for a rare‑disease therapy, undermining prior optimism and clouding the drug’s development pathway. The mixed late‑stage results force a strategic reassessment, cooling investor expectations and leaving researchers and patients waiting for next steps or alternative approaches.