The FDA is expected to decide on Voxzogo for Hypochondroplasia by May 30, 2027, under an sNDA from BioMarin.
What the FDA is deciding on Voxzogo for Hypochondroplasia
The FDA is reviewing BioMarin's request to approve Voxzogo (vosoritide) for a new use, hypochondroplasia.
BioMarin asks the FDA to add hypochondroplasia to Voxzogo's label. MedlinePlus describes hypochondroplasia as a form of short stature similar to but milder than achondroplasia, with most cases caused by changes in the same gene, FGFR3. BioMarin calls Voxzogo the first potential targeted therapy for it.
The European Medicines Agency has also validated BioMarin's application. Voxzogo is already approved for children with achondroplasia, where a separate decision on full approval is due February 28, 2027.
How Voxzogo works
Voxzogo: c-type natriuretic peptide (CNP) analog that binds the NPR-B receptor and dampens the signaling switched on by the overactive FGFR3 receptor, which lets the growth plates make bone faster. Our full Voxzogo profile covers how it works, side effects in plain language, and its trial history.
What an approval or a rejection of Voxzogo 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. The letter describes what the FDA wants addressed, and the company then decides whether to resubmit, run more studies or stop. Either way, this page updates with the outcome and what it means.
Frequently asked questions
When will the FDA decide on Voxzogo?
The FDA's target date (PDUFA date) for Voxzogo in Hypochondroplasia is May 30, 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.
What is Voxzogo being reviewed for?
BioMarin submitted an sNDA for Voxzogo in Hypochondroplasia.
What to know: BioMarin asks the FDA to add hypochondroplasia to Voxzogo's label. MedlinePlus describes hypochondroplasia as a form of short stature similar to but milder than achondroplasia, with most cases caused by changes in the same gene, FGFR3. BioMarin calls Voxzogo the first potential targeted therapy for it. The European Medicines Agency has also validated BioMarin's application. Voxzogo is already approved for children with achondroplasia, where a separate decision on full approval is due February 28, 2027.
How it works: C-type natriuretic peptide (CNP) analog that binds the NPR-B receptor and dampens the signaling switched on by the overactive FGFR3 receptor, which lets the growth plates make bone faster.
More: the Voxzogo drug page explains it in plain language.
What did the Voxzogo trials show?
Accelerated approval rested on Study 1 (NCT03197766), which randomly assigned 121 children aged 5.1-14.9 with achondroplasia to daily Voxzogo (60 children) or placebo (61) for 52 weeks. Children on Voxzogo grew 1.57cm a year more than children on placebo (95% CI 1.22 to 1.93), and their height compared with other children with achondroplasia also improved.
More: the Voxzogo drug page has the full trial history and sources.
What are the side effects of Voxzogo?
Voxzogo has no boxed warning and no contraindications. Its one warning is about low blood pressure.
Most common: Injection site redness (75% against 69% on placebo), Injection site swelling (62% against 36%), Vomiting (27% against 20%), Injection site hives (25% against 10%), Joint pain (15% against 7%), Lower blood pressure (13% against 5%).
More: the Voxzogo drug page has the label's warnings in full.
How much will Voxzogo cost?
Voxzogo is already sold in the US for other conditions. Trial Friend does not list a price, and coverage for Hypochondroplasia depends on the FDA decision and your insurer.
Drugmaker help: BioMarin RareConnections.
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 Q3 2026 results, November 2, 2026. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.