BioMarin suffers another blow to rare disease portfolio in Phase 3 flop
BioSpace
BioMarin's investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
pharminent
May 19, 2026 (Last updated: May 19, 2026)
BioSpace
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.