San Rafael drugmaker BioMarin is getting another big moment in front of federal regulators. The company says the U.S. Food and Drug Administration has accepted its supplemental new drug application (sNDA) to convert VOXZOGO® (vosoritide) from accelerated approval to traditional full approval for children with achondroplasia whose growth plates are still open. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027, and BioMarin says the filing leans on long-term safety and efficacy results plus new adult-height data from ongoing extension studies.
In a press release distributed through PR Newswire, BioMarin said the FDA has formally accepted the sNDA and confirmed the Feb. 28, 2027 decision date. VOXZOGO first received accelerated approval in 2021 and, according to the company, is approved in Japan and Australia and available in more than 50 countries. The FDA has described that 2021 decision as based on improved annualized growth velocity in children with achondroplasia.
Long-Term Data And What Is In The Filing
For this new application, BioMarin says it has pulled together adult-height outcomes plus long-term safety and efficacy findings from extension studies 111-205, 111-208 and 111-302. Those trials look at growth, arm span, bone health and a range of other clinical measures over time. At recent medical meetings, the company presented extended follow-up showing mean standing-height gains of about 10.6 centimeters after six years and roughly 13.6 centimeters after eight years in children who started VOXZOGO after age five, compared with untreated natural-history cohorts. BioMarin has presented those numbers as part of the evidence package behind the sNDA.
Regulatory Context
VOXZOGO entered the market under the FDA’s accelerated-approval pathway in 2021, cleared on the basis of a surrogate endpoint, annualized growth velocity, rather than final adult-height outcomes. That pathway requires follow-up data to confirm that the surrogate translates into real clinical benefit. FDA approval documents spell out that continued marketing could depend on adult-height results and other long-term outcomes, and that failure to confirm benefit can put the approval at risk of withdrawal. BioMarin says the new adult-height data and extended follow-up in the sNDA are intended to meet those postmarketing expectations, and FDA letters detail the confirmatory-study requirements the company is expected to satisfy.
What Families And Clinicians Should Know
BioMarin notes that VOXZOGO is already in routine clinical use around the world, and the company promotes a slate of patient-support offerings, including clinical coordinators, a caregiver-mentorship program and a U.S. physician directory. In the United States, families can reach RareConnections case managers at 1-833-VOXZOGO (1-833-869-9646). The treatment is given as a daily subcutaneous injection, and trial reports have flagged potential side effects such as transient drops in blood pressure that call for monitoring and careful shared decision-making. For now, families and providers are still relying on current safety and efficacy data while the FDA reviews the sNDA, and the outcome of that review is expected to help shape labeling and long-term treatment guidance. PR Newswire and BioMarin’s published materials include prescribing information, safety details and support resources for families and clinicians.
The FDA review is expected to run through the PDUFA timeline, with a decision slated by Feb. 28, 2027, on whether VOXZOGO’s U.S. marketing status moves from accelerated to full approval. In the meantime, families and specialty clinicians are being encouraged to stay connected with centers that regularly treat achondroplasia and with BioMarin’s support programs while regulators work through the data.
