BioMarin's rare disease therapy BMN 401 failed to show a meaningful clinical benefit in its Phase 3 ENERGY 3 trial in children with ENPP1 deficiency, a serious genetic condition affecting about 1 in 200,000 people.
The trial met one co‑primary endpoint (a statistically significant increase in plasma inorganic pyrophosphate, or PPi), but missed the other co‑primary endpoint, which was improvement in radiographic skeletal healing (RGI‑C scores).
No positive trends were seen on secondary endpoints such as rickets severity scores and growth Z‑scores, leading BioMarin to conclude that the biomarker gains did not translate into meaningful clinical improvements.
BMN 401 was generally well‑tolerated with no new safety signals, and BioMarin is now evaluating the data to determine next steps while cautioning that approval prospects are uncertain.
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