Voxzogo (vosoritide)
An approved treatment for Achondroplasia.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Vosoritide wherever you encounter it, plus the key facts at a glance.
- Generic name
- Vosoritide
- Brand name
- Voxzogo
- Drug class
- C-type natriuretic peptide (CNP) analog
- Manufacturer
- BioMarin Pharmaceutical
- How it's taken
- Given once a day as an injection under the skin, at about the same time each day, by a caregiver after training.
A once-daily injection under the skin, given at home by a trained caregiver, and the first drug the FDA approved for achondroplasia. It received accelerated approval on November 19, 2021 for children 5 and older, and on October 20, 2023 the FDA extended it to children of any age whose growth plates are still open. In its main trial, children aged 5-14 grew 1.57cm a year more than children on placebo.
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Where Vosoritide fits
The first FDA-approved drug for achondroplasia and the only one approved from birth. Yuviwel (navepegritide), approved in February 2026 for children 2 and older, is a weekly CNP drug, and infigratinib, a daily pill, has an FDA decision due February 4, 2027. No trial has compared these drugs with each other.
How Vosoritide works
Achondroplasia is caused by a change in the FGFR3 gene that makes the FGFR3 receptor overactive, and that overactivity slows the growth plates, the zones of cartilage near the ends of long bones where growth happens. CNP is a natural signal that pushes back by binding a receptor called NPR-B and dampening the pathway FGFR3 switches on. Vosoritide is a lab-made version of CNP with 2 extra building blocks that slow its breakdown in the body, and it is injected once a day.
Side effects and safety
- Low blood pressure. Brief drops in blood pressure were seen in the trials. Eat a meal and drink 240-300mL of fluid in the hour before each dose, and call the doctor about dizziness, tiredness or nausea.
- Kidney impairment. Not recommended with an eGFR below 60.
- Weight, growth and development checked every 3 to 6 months, with the dose adjusted to weight
- Treatment stops when the growth plates close and no further growth is possible
- A meal and 240-300mL of fluid in the hour before every dose
Voxzogo has no boxed warning and no contraindications. Its one warning is about low blood pressure. In the main trial, 8 of 60 children on Voxzogo (13%) had brief drops in blood pressure against 3 of 61 on placebo (5%), usually starting about half an hour after the shot and passing within about half an hour. Eating and drinking 240-300mL of fluid in the hour before each dose lowers this risk.
Injection site reactions were common in both groups, 85% on Voxzogo and 82% on placebo, though children on Voxzogo had about 4 times as many reaction events over the year.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Vosoritide
Given once a day as an injection under the skin, at about the same time each day, by a caregiver after training. The dose is based on body weight, from 0.096mg for a baby weighing 3kg up to 0.8mg for anyone weighing 90kg or more, and it is mixed fresh from powder for each dose. A missed dose can be given within 12 hours, and after that it is skipped and the next dose given as usual. Vials can be kept at room temperature for up to 90 days.
Availability and cost
Only available as the brand-name product.
Help paying for Voxzogo
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
Case and Field Reimbursement Managers help with the insurance process, coverage options, insurance changes and getting the prescription to the specialty pharmacy.
The official page does not say who qualifies. Ask the program. · source - Copay help
BioMarin Co-Pay Assistance Program for eligible patients with commercial prescription insurance and a prescription for an FDA-approved indication. Not valid with Medicare, Medicaid, VA, DoD, TRICARE or other government insurance. BioMarin says over 96% of eligible commercially insured families paid $0 out of pocket.
For: private insurance · source
Good to know: Reach a case manager at 1-833-VOXZOGO (1-833-869-9646). The official page does not describe a free medicine program for uninsured patients; ask the case manager. Some state residents may not be eligible for all program elements.
- From a charity · Little People of AmericaGrant for Overall Accessible Living (GOAL) fundOpen
Pays for: Mobility devices and occupational tools (partial cost; LPA membership required).
The foundation says: “Applications are being accepted for the GOAL Fund.”
Access and eligibility
Approved for children of any age with achondroplasia whose growth plates are still open, with doses starting at a body weight of 3kg. It is not recommended with an eGFR below 60. Children with significant heart or blood vessel disease, or on blood pressure medicine, were left out of the trials.
Source: BioMarin RareConnections (Voxzogo financial assistance)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
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.
Upper-to-lower body proportions did not differ from placebo over that year[1]. A second placebo-controlled study in 64 children aged 4.4 months to under 5 supported use in younger children, with a safety profile similar to older children[1].
Development history
BioMarin developed vosoritide and received FDA accelerated approval on November 19, 2021 for children 5 and older with open growth plates, the first approval for achondroplasia. On October 20, 2023 the FDA extended the approval to children of all ages[2]. Continued approval depends on confirming clinical benefit. On July 13, 2026 BioMarin said the FDA accepted its application for full approval, based on long-term data from 3 ongoing studies including adult height, with a decision date of February 28, 2027[3].
Explore Achondroplasia trials
Other Achondroplasia treatments
Achondroplasia has 2 FDA-approved growth treatments, both CNP analogs under accelerated approval: Voxzogo (vosoritide), a daily injection approved November 19, 2021 and now covering all ages with open growth plates, and Yuviwel (navepegritide), a weekly injection approved February 27, 2026 for children 2 and older. Both are used only while growth plates are open.
Common questions about Vosoritide
▸What is Vosoritide (Voxzogo)?
A once-daily injection under the skin, given at home by a trained caregiver, and the first drug the FDA approved for achondroplasia. It received accelerated approval on November 19, 2021 for children 5 and older, and on October 20, 2023 the FDA extended it to children of any age whose growth plates are still open. In its main trial, children aged 5-14 grew 1.57cm a year more than children on placebo.
▸How does Vosoritide work?
Achondroplasia is caused by a change in the FGFR3 gene that makes the FGFR3 receptor overactive, and that overactivity slows the growth plates, the zones of cartilage near the ends of long bones where growth happens. CNP is a natural signal that pushes back by binding a receptor called NPR-B and dampening the pathway FGFR3 switches on. Vosoritide is a lab-made version of CNP with 2 extra building blocks that slow its breakdown in the body, and it is injected once a day.
▸What are the side effects of Vosoritide?
Voxzogo has no boxed warning and no contraindications. Its one warning is about low blood pressure. In the main trial, 8 of 60 children on Voxzogo (13%) had brief drops in blood pressure against 3 of 61 on placebo (5%), usually starting about half an hour after the shot and passing within about half an hour. Eating and drinking 240-300mL of fluid in the hour before each dose lowers this risk. Injection site reactions were common in both groups, 85% on Voxzogo and 82% on placebo, though children on Voxzogo had about 4 times as many reaction events over the year.
▸How is Vosoritide taken?
Given once a day as an injection under the skin, at about the same time each day, by a caregiver after training. The dose is based on body weight, from 0.096mg for a baby weighing 3kg up to 0.8mg for anyone weighing 90kg or more, and it is mixed fresh from powder for each dose. A missed dose can be given within 12 hours, and after that it is skipped and the next dose given as usual. Vials can be kept at room temperature for up to 90 days.
▸Is Vosoritide FDA approved?
Yes, Vosoritide (Voxzogo) is FDA approved (2021) for the treatment of Achondroplasia.
▸Why does my child need to eat and drink before each Voxzogo shot?
It lowers the chance of a drop in blood pressure. The label asks for a meal and about 240-300mL of fluid in the hour before each dose, because brief blood pressure drops were seen in the trials, usually within about half an hour of the shot.
▸When will the FDA decide on full approval for Voxzogo?
BioMarin says the FDA set a decision date of February 28, 2027 for converting Voxzogo's 2021 accelerated approval to full approval. Voxzogo stays available under its accelerated approval during the review.
▸Does Voxzogo increase final adult height?
That has not been confirmed yet. The FDA approved Voxzogo based on faster yearly growth, and BioMarin's full-approval application includes adult height data from ongoing studies, which the FDA is reviewing ahead of its February 28, 2027 decision date.
▸What is the difference between Voxzogo and Yuviwel?
Both are CNP drugs that counter the overactive FGFR3 signal in achondroplasia. Voxzogo is injected once a day and is approved for children of any age with open growth plates, while Yuviwel is injected once a week and is approved from age 2. No trial has compared them, so there is no evidence that one works better than the other.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2025-11-25. VOXZOGO (vosoritide) for injection, for subcutaneous use: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=228e8560-04a4-4bb1-a81f-29531a9e4d27
- U.S. Food and Drug Administration. Drugs@FDA: VOXZOGO approval history (NDA 214938). https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214938
- BioMarin · 2026-07-13. U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO (vosoritide) for Children With Achondroplasia. https://www.biomarin.com/news/press-releases/u-s-food-and-drug-administration-accepts-biomarins-supplemental-new-drug-application-for-full-approval-of-voxzogo-vosoritide-for-children-with-achondroplasia/
- ClinicalTrials.gov. A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of BMN 111 in Children With Achondroplasia. https://clinicaltrials.gov/study/NCT03197766
- BioMarin. Patient Assistance Program: Insurance and Co-Pay Support. https://www.voxzogo.com/en-us/financial-assistance/