Biomarin Pharmaceutical Inc BMRN
Biomarin Pharmaceutical Inc (Nasdaq: BMRN) is a rare genetic drug developer. Its lead program, VOXZOGO (vosoritide), is in Phase 3 for achondroplasia; hypochondroplasia (sNDA). Its next catalyst on record is VOXZOGO: PDUFA target action date for sNDA (full approval, achondroplasia) (February 28, 2027).
Upcoming catalysts
- February 28, 2027VOXZOGO — PDUFA target action date for sNDA (full approval, achondroplasia)
In July 2026, we announced that the FDA accepted our 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.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| VOXZOGO | achondroplasia (sNDA for full approval under review) | protein/peptide | Approved |
| GALAFOLD | Fabry disease (via Amicus acquisition) | small molecule | Approved |
| POMBILITI + OPFOLDA | Pompe disease (via Amicus acquisition) | biologic-other | Approved |
| VOXZOGO | hypochondroplasia (sNDA submitted July 2026) | protein/peptide | Phase 3 |
| BMN 333 | long-acting C-type natriuretic peptide, skeletal conditions | protein/peptide | Phase 2 |
Recent results and decisions
- Aug 31, 2026completed BioMarin completed acquisition of Alesta Therapeutics B.V. on August 31, 2026.
In connection with its acquisition of Alesta Therapeutics B.V. (Alesta), a Dutch private limited liability company (besloten vennootschap met beperkte aansprakelijkheid), which was completed on August 31, 2026
Filing - Aug 6, 2026submitted BioMarin recently submitted an sNDA to FDA for VOXZOGO in hypochondroplasia; potential 2027 launch, application status update planned with Q3 earnings.
BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia.
Filing - Aug 6, 2026accepted FDA accepted the sNDA for full approval of VOXZOGO in children with achondroplasia, PDUFA date February 28, 2027 (announced in July).
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.
Filing
4 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.