FDA Accepts VOXZOGO sNDA; PDUFA Feb. 28, 2027
Decision brief
Answer first · skim in under a minute
FDA accepted BioMarin's VOXZOGO sNDA to convert accelerated achondroplasia approval to full; PDUFA target action date is Feb. 28, 2027.
Key questions this brief answers
- What did FDA accept for VOXZOGO?
- What data support the full-approval sNDA?
- Is VOXZOGO already approved in the U.S.?
FDA accepts VOXZOGO sNDA full approval filing from BioMarin to convert the 2021 accelerated achondroplasia indication to traditional approval. The company said on July 13, 2026 that FDA set a PDUFA target action date of Feb. 28, 2027.
Contents9 sections
Key Takeaways
- FDA accepted BioMarin's sNDA seeking full approval of VOXZOGO (vosoritide) in pediatric achondroplasia.
- PDUFA target action date: Feb. 28, 2027.
- Package draws on long-term data from studies 111-205, 111-208, and 111-302, including adult height outcomes.
- U.S. label remains under accelerated approval until FDA acts on the confirmatory package.
VOXZOGO sNDA at a glance
| Field | Detail |
|---|---|
| Product | VOXZOGO (vosoritide), CNP analog |
| Company | BioMarin Pharmaceutical Inc. |
| FDA action | sNDA acceptance announced July 13, 2026 |
| Goal | Convert accelerated approval to full approval in achondroplasia |
| PDUFA date | Feb. 28, 2027 |
| Evidence base | Studies 111-205, 111-208, 111-302 (long-term growth and skeletal measures) |
| Prior U.S. status | Accelerated approval since 2021 (annualized growth velocity) |
What did the FDA accept?
BioMarin announced FDA acceptance of an sNDA for VOXZOGO seeking full approval in children with achondroplasia. If approved, BioMarin said VOXZOGO would be the first achondroplasia therapy to convert from accelerated to traditional approval based on a comprehensive package that includes adult height and other long-term clinical measures.
See BioMarin's July 13 PR Newswire sNDA acceptance release.
What evidence is in the package?
BioMarin said the sNDA is supported by substantial long-term safety and efficacy data from three ongoing studies — 111-205, 111-208, and 111-302 — including clinically meaningful growth results and improvements in skeletal measures such as proportionality and arm span. The company described the package as the longest efficacy and safety dataset of any medicine studied in achondroplasia.
The original registrational program for vosoritide in achondroplasia is tracked on ClinicalTrials.gov; see NCT03197766 (vosoritide Phase 3 in achondroplasia) for the core randomized evidence base that underpinned accelerated approval.
Why convert from accelerated approval?
VOXZOGO received FDA accelerated approval in 2021 based on improvement in annualized growth velocity, a surrogate reasonably likely to predict clinical benefit. Continued approval may be contingent on confirmatory evidence. BioMarin said this sNDA is intended to fulfill the postmarketing requirement using ongoing open-label extension data versus available natural history.
FDA's general accelerated-approval framework is summarized on the FDA accelerated approval pathway page.
What is achondroplasia and how does VOXZOGO work?
Achondroplasia is the most common skeletal dysplasia causing disproportionate short stature, driven by FGFR3 gain-of-function that slows endochondral ossification. Worldwide incidence is about one in 25,000 live births. VOXZOGO is a C-type natriuretic peptide analog that positively regulates signaling downstream of FGFR3 to promote endochondral bone growth. BioMarin said more than 5,000 infants and children in more than 50 countries have been treated since the 2021 approval.
What should clinicians watch through the PDUFA date?
Until Feb. 28, 2027, U.S. labeling remains under accelerated approval. Watch for FDA information requests, any advisory committee, and how adult-height and proportionality endpoints are described in a potential traditional label. Access support continues through BioMarin's RareConnections program.
Related coverage: VOXZOGO long-term achondroplasia data, prior label-expansion analysis, and other recent rare-disease FDA filing news.
Frequently Asked Questions
What did FDA accept for VOXZOGO?
BioMarin said the FDA accepted its supplemental New Drug Application seeking full (traditional) approval of VOXZOGO (vosoritide) for children with achondroplasia. The PDUFA target action date is Feb. 28, 2027.
What data support the full-approval sNDA?
The sNDA is supported by long-term safety and efficacy data from three ongoing studies (111-205, 111-208, and 111-302), including adult height and other skeletal outcomes such as body proportionality and arm span over extended follow-up.
Is VOXZOGO already approved in the U.S.?
Yes. VOXZOGO received FDA accelerated approval in 2021 to increase linear growth in children with achondroplasia and open epiphyses, based on annualized growth velocity. Continued approval may depend on confirmatory evidence; this sNDA is intended to fulfill that postmarketing requirement.
