BMN 401 — NDA/BLA submission
2H'26
Regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-disease launch in 2027.Source filing
BMRN biotech catalysts & pipeline
BIOMARIN PHARMACEUTICAL INC · Data as of 2026-10-25
Upcoming catalysts
BMN 333 — Interim data
in 2027
The Phase 2/3 study is enrolling, with a data update expected in 2027.Source filing
VOXZOGO (vosoritide) — PDUFA
Feb 28
An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span, is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.Source filing
BMN 820 — Clinical data readout
in 2028
The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028.Source filing