BioMarin Pharmaceutical Inc. BMRN
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) is a rare genetic drug developer. Its lead program, VOXZOGO sNDA for hypochondroplasia (HCH), is in Phase 3 for Hypochondroplasia. Its next catalyst on record is VOXZOGO: PDUFA target action date for full approval in achondroplasia (February 2027).
Upcoming catalysts
- February 2027VOXZOGO — PDUFA target action date for full approval in achondroplasia
the FDA accepted our sNDA for full approval of VOXZOGO in children with achondroplasia, with a PDUFA target action date of February 28, 2027
- May 2027VOXZOGO — PDUFA target action date for hypochondroplasia sNDA
the U.S. Food and Drug Administration (FDA) set a Prescription Drug User Fee Act (PDUFA) target action date of May 30, 2027 for supplemental new drug application (sNDA) to expand the use of VOXZOGO for the treatment of hypochondroplasia (HCH)
Next further out: VOXZOGO — EMA decision on hypochondroplasia approval (date not stated). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| VOXZOGO | Achondroplasia | protein/peptide | Approved |
| VIMIZIM | MPS IVA | protein/peptide | Approved |
| NAGLAZYME | MPS VI | protein/peptide | Approved |
| PALYNZIQ | Phenylketonuria (PKU) | protein/peptide | Approved |
| GALAFOLD | Fabry disease | small molecule | Approved |
| BRINEURA | CLN2 disease | protein/peptide | Approved |
| ALDURAZYME | MPS I | protein/peptide | Approved |
| POMBILITI + OPFOLDA | Pompe disease | protein/peptide | Approved |
| VOXZOGO sNDA for hypochondroplasia | Hypochondroplasia | protein/peptide | Phase 3 |
| VOXZOGO sNDA for full approval in achondroplasia | Achondroplasia | protein/peptide | Phase 3 |
| BMN 450 (HM15421/GC1134A) | Fabry disease | protein/peptide | Pre-clinical |
| BMN 440 (formerly ALE1) | Hypophosphatasia (HPP) | small molecule | Pre-clinical |
Recent results and decisions
- Nov 2, 2026submitted FDA set PDUFA action date of May 30, 2027 for VOXZOGO supplemental NDA for hypochondroplasia.
The U.S. Food and Drug Administration (FDA) has set a PDUFA target action date of May 30, 2027 for its supplemental NDA to expand the use of VOXZOGO for the treatment of HCH, the first potential targeted therapy for the condition.
Filing - Nov 2, 2026accepted European Medicines Agency validated VOXZOGO submission for hypochondroplasia.
BioMarin also announced today that the European Medicines Agency (EMA) validated its submission for the approval of VOXZOGO to treat HCH.
Filing - positive Five-year PROPEL extension data showed durable motor and pulmonary function outcomes for POMBILITI + OPFOLDA in Pompe disease.
In adults with late-onset Pompe disease, POMBILITI + OPFOLDA demonstrated durable motor function outcomes and relative stabilization of pulmonary function.
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.