BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency
BioMarin's pivotal Phase 3 ENERGY 3 trial for BMN 401 in children with ENPP1 deficiency met only one of two co-primary endpoints. While the drug successfully increased plasma inorganic pyrophosphate (PPi) levels, it failed to show improvement in Radiographic Global Impression of Change (RGI-C) scores, the key clinical measure of rickets severity.
Key figures
- Drug Name
- BMN 401
- Phase Of Trial
- Phase 3
- Age range
- 1-12 years
- Study name
- ENERGY 3
- Participants
- 27
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