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Monday, May 18, 2026

BioMarin pivotal Phase 3 trial of BMN 401 meets biomarker endpoint but misses key clinical endpoint

 

BioMarin pivotal Phase 3 ENERGY 3 trial of BMN 401 meets biomarker endpoint but misses key clinical endpoint in children with ENPP1 deficiency

  • Company says it will evaluate next steps for the BMN 401 program following mixed ENERGY 3 results.
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