BioMarin's Phase 3 trial for its investigational therapy in ENPP1 deficiency failed to show clinical improvements, missing key secondary endpoints related to rickets severity and growth. This setback raises concerns about the future of their rare disease portfolio.
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.
Original title: “BioMarin suffers another blow to rare disease portfolio in Phase 3 flop”