Voxzogo (vosoritide) for Achondroplasia (conversion to full approval)
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The FDA is expected to decide on Voxzogo for Achondroplasia (conversion to full approval) by February 28, 2027, under a sNDA from BioMarin.
What is being decided
BioMarin has a sNDA under FDA review for Voxzogo (vosoritide) in Achondroplasia (conversion to full approval). Application to convert the 2021 accelerated approval into full approval.
Who this decision matters to
Achondroplasia is the most common form of short-limbed dwarfism, caused by a mutation in the FGFR3 gene that disrupts the conversion of cartilage to bone in the growth plates. This results in disproportionate short stature with an average-size trunk but shortened arms and legs. About 80% of cases arise from new spontaneous mutations rather than being inherited from an affected parent.
Prevalence: Approximately 1 in 25,000 live births; estimated 25,000 to 30,000 people in the United States. See our full Achondroplasia page for current treatments, recruiting trials, and community resources.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Achondroplasia treatment stands today
Read our full Achondroplasia treatment summary
Clinical trials for achondroplasia are exploring multiple strategies to promote bone growth and address the complications of FGFR3 overactivation. Voxzogo (vosoritide), a daily injection approved in November 2021, established the path for growth-promoting therapies, and in February 2026 the FDA approved Yuviwel (navepegritide), a once-weekly CNP injection for children 2 and older whose growth plates are still open.
Both received accelerated approval based on faster yearly growth, and continued approval of each depends on confirmatory trials. On October 6, 2026 the FDA accepted BridgeBio's application for infigratinib, a once-daily pill that blocks FGFR3, for children with achondroplasia, with a decision due February 4, 2027.
Gene therapy and combination treatments are in earlier development. Trials measure treatment response through growth velocity, body proportions, and functional outcomes like mobility and respiratory function.
For children, early enrollment in growth-promoting trials may maximize benefit while growth plates remain open. Adults with achondroplasia may benefit from trials addressing spinal stenosis, sleep apnea, and pain management.
Your geneticist, orthopedic specialist, or skeletal dysplasia clinic can help evaluate whether current trials are appropriate. Little People of America and the MAGIC Foundation maintain information on clinical trials and connect families with specialized care centers.
The Achondroplasia page has every treatment and trial →Meanwhile, 11 Achondroplasia trials are recruiting
Whatever the FDA decides here, research on Achondroplasia does not stop. A few currently enrolling studies, US sites first:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Achondroplasia. Unsubscribe anytime.
Our coverage
Frequently asked questions
When will the FDA decide on Voxzogo?
The FDA's target date (PDUFA date) for Voxzogo in Achondroplasia (conversion to full approval) is February 28, 2027.
Who disclosed it: BioMarin. The FDA does not publish these dates.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
Source: BioMarin press release
What is Voxzogo being reviewed for?
BioMarin submitted a sNDA for Voxzogo in Achondroplasia (conversion to full approval).
What to know: Application to convert the 2021 accelerated approval into full approval.
Is there already an FDA-approved treatment for Achondroplasia?
Yes. Trial Friend's drug list includes 1 FDA-approved drug for Achondroplasia.
Approved: Yuviwel (navepegritide).
Check: the Achondroplasia page also searches FDA drug labels for approved treatments.
Can I get Voxzogo before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 11 Achondroplasia trials are recruiting now. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask BioMarin directly. Registered programs are listed on ClinicalTrials.gov.
How much will Voxzogo cost?
BioMarin has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Voxzogo decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from BioMarin press release. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.