FDA accepts sNDA for vosoritide in children with achondroplasia

The FDA has accepted a supplemental new drug application (sNDA) from BioMarin Pharmaceutical Inc seeking full approval of vosoritide (Voxzogo) in children with achondroplasia, the company announced.¹ The agency has set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.¹

If granted, the conversion would mark the first time a therapy for achondroplasia has moved from accelerated to traditional approval on the strength of a comprehensive long-term clinical data package. "This submission for VOXZOGO is supported by the largest body of evidence for any medicine in achondroplasia, reflecting BioMarin's long-standing commitment to advancing the science of skeletal growth," said Greg Friberg, MD, executive vice president and chief research & development officer at BioMarin, in the company's announcement. "The clinical data demonstrate meaningful improvements across multiple skeletal growth-related measures beyond annualized growth velocity in children with this condition."¹

Vosoritide sNDA data and regulatory pathway in achondroplasia

The sNDA draws on long-term safety and efficacy data from 3 ongoing studies (111-205, 111-208, and 111-302), which the company describes as the longest efficacy and safety follow-up generated for any medicine studied in achondroplasia.¹ The submission includes adult height outcomes along with additional measures collected over extended follow-up, including body proportionality and arm span, intended to extend beyond the annualized growth velocity endpoint that supported the original accelerated approval.¹

Vosoritide first received FDA accelerated approval in November 2021 for children aged 5 years and older with achondroplasia and open growth plates, based on a 52-week, randomized, placebo-controlled phase 3 trial in 121 children that showed a 1.57 cm/year improvement in annualized growth velocity over placebo.² The indication was later expanded, in October 2023, to include children of all ages with open epiphyses, incorporating phase 2 data in children younger than 5 years.³ Because accelerated approval is granted on a surrogate or intermediate endpoint reasonably likely to predict clinical benefit, continued marketing has remained contingent on confirmatory evidence—the requirement this sNDA is intended to satisfy.¹

Disease burden and current treatment landscape

Achondroplasia is the most common skeletal dysplasia causing disproportionate short stature, arising from an activating mutation in the FGFR3 gene that impairs endochondral ossification in the long bones, spine, face, and skull base.¹ The condition occurs in roughly 1 in 25,000 live births worldwide, and more than 80% of affected children are born to average-stature parents as the result of a spontaneous mutation.¹ Vosoritide is currently the only approved medicine for achondroplasia usable from birth, and BioMarin states it has been used by more than 5,000 infants and children across more than 50 countries.¹ Roughly a quarter of people with achondroplasia have open growth plates and would be eligible candidates for growth-promoting therapy during childhood.¹

Mechanism and prior evidence base

Vosoritide is a C-type natriuretic peptide analog that binds natriuretic peptide receptor-B, activating a signaling cascade that counteracts the overactive FGFR3 pathway responsible for impaired chondrocyte proliferation in achondroplasia.² Open-label extension data have shown sustained growth-velocity gains through several years of continued dosing, and the most frequently reported adverse effects across the development program have been injection-site reactions and transient hypotension, which the label recommends mitigating with food and fluid intake before dosing.²

Interpretation and remaining questions

The data package BioMarin has submitted moves beyond growth velocity—a surrogate measure—toward outcomes with more direct relevance to long-term skeletal health, such as body proportionality and arm span, alongside adult height.¹ Whether this evidence is sufficient for the FDA to convert the indication to traditional approval will depend on the agency's review of durability and clinical meaningfulness of these endpoints, which has not yet been made public in granular form. Adult height data in particular can take many years to mature, and how the agency weighs surrogate versus more patient-relevant proportionality measures in a rare pediatric disease remains an open regulatory question.

Limitations and next steps

The clinical findings referenced in the announcement come from open-label extension studies rather than newly randomized, controlled comparisons, and the company has not yet disclosed full efficacy figures from studies 111-205, 111-208, and 111-302 in peer-reviewed form. The FDA's review is ongoing, with a target decision date of February 28, 2027; the agency's action could include additional information requests, label changes, or continued accelerated status rather than a full conversion.¹ Clinicians and families should await formal FDA action and peer-reviewed publication of the underlying long-term data before treatment decisions are altered based on this announcement.

References

1. BioMarin Pharmaceutical Inc. US Food and Drug Administration accepts BioMarin's supplemental New Drug Application for full approval of VOXZOGO (vosoritide) for children with achondroplasia. News release. July 13, 2026. Accessed July 13, 2026. https://investors.biomarin.com/news/news-details/2026/U-S--Food-and-Drug-Administration-Accepts-BioMarins-Supplemental-New-Drug-Application-for-Full-Approval-of-VOXZOGO-vosoritide-for-Children-With-Achondroplasia/default.aspx

2. US Food and Drug Administration. VOXZOGO (vosoritide) prescribing information. Revised 2024. Accessed July 13, 2026. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/214938s004lbl.pdf

3. Healio. FDA expands indication for vosoritide to include children of all ages with achondroplasia. October 23, 2023. Accessed July 13, 2026. https://www.healio.com/news/endocrinology/20231023/fda-expands-indication-for-vosoritide-to-include-children-of-all-ages-with-achondroplasia