BMN 351 — Topline data
2H'26
The 12 mg/kg dose cohort continues to enroll participants, with topline data readout from this cohort expected in 2H'26.Source filing
BMRN biotech catalysts & pipeline
BIOMARIN PHARMACEUTICAL INC · Data as of 2026-09-08
Upcoming catalysts
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
VOXZOGO (vosoritide) — Filing acceptance
Q3 2026
The company expects to be notified of sNDA acceptance by Q3 2026.Source filing
VOXZOGO (vosoritide) — NDA/BLA submission
2H'26
Regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-class launch in 2027.Source filing
BMN 333 — Interim data
in 2027
The Phase 2/3 study is enrolling, with a data update expected in 2027.Source filing
VOXZOGO (vosoritide) — Regulatory decision
2027
If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.Source filing
VOXZOGO (vosoritide) — PDUFA
Feb 28
In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.Source filing
BMN 820 — Topline data
in 2028
The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028.Source filing
VOXZOGO (vosoritide) — Conference presentation
Sep 8–Sep 10
The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September.Source filing