Vosoritide
C-type natriuretic peptide (CNP) analogue
Status
Approved
Approved in the US in November 2021 under accelerated approval and in the EU in August 2021; the US indication now covers children of any age with achondroplasia whose growth plates are still open, and the EU indication covers patients from 4 months of age. BioMarin's application to convert the US accelerated approval into full approval was accepted in July 2026 with a target decision date of 28 February 2027, and a separate application to add hypochondroplasia was submitted in September 2026.
Evidence
Moderate
One 121-child randomised phase 3 trial in achondroplasia with open-label extensions, supported by a second randomised trial in children under 5 and a 2026 randomised trial in hypochondroplasia.
A laboratory version of C-type natriuretic peptide, given as a daily injection to increase growth in children with achondroplasia, the most common form of dwarfism, while their growth plates are still open.
Structure
39 residuesPoint at a bead, or focus the chain and use the arrow keys, to name it. Pick a chemistry to highlight it.
Sequence as text
H-P-G-Q-E-H-P-N-A-R-K-Y-K-G-A-N-K-K-G-L-S-K-G-C-F-G-L-K-L-D-R-I-G-S-M-S-G-L-G-C-OH
What it is used for
- Achondroplasia in children whose epiphyses are still open (approved)
- Hypochondroplasia, where a phase 3 trial reported a 2.33 cm per year gain in growth velocity and regulatory submissions followed in 2026 (studied)
How it works
Achondroplasia is caused by a gain-of-function change in the receptor FGFR3, which puts a constant brake on the cartilage cells in the growth plate. Vosoritide binds the natriuretic peptide receptor B, raising cyclic GMP inside those cells and inhibiting the MAPK pathway downstream of FGFR3 at the level of RAF-1. That releases the brake, so cartilage cells multiply and mature normally and the bone grows. The effect depends on open growth plates.
How strong the evidence is
Moderate
The pivotal 52-week trial randomised 121 children aged 5 to 14 years with genetically confirmed achondroplasia to daily vosoritide or placebo and measured annualised growth velocity, with the gain sustained in open-label extensions running several years. A second randomised trial covered children from 4.4 months to under 5 years. In hypochondroplasia, the phase 3 trial published in NEJM Evidence in September 2026 reported 2.33 cm per year more growth than placebo, 2.35 cm more standing height and a height Z-score gain of 0.39. Final height and functional benefit are still being confirmed.
Risks and side effects
- Transient drops in blood pressure, usually within about half an hour of a dose, which is why food and fluid are advised beforehand
- Injection site reactions are very common, including redness, swelling and hives, and two children in the pivotal trial stopped treatment because of injection pain and anxiety
- Vomiting, arthralgia, diarrhoea, dizziness and raised alkaline phosphatase were more frequent than on placebo
- Not recommended when kidney function is reduced below an eGFR of 60 mL/min/1.73 m2
- Anti-drug antibodies develop in a third or more of treated children, so far without effect on safety or growth
- Approval is accelerated and based on growth velocity, an intermediate measure, with confirmation of long-term benefit still pending
Label dosing
Label: one subcutaneous injection daily, with the dose set by the child's actual body weight and treatment stopped permanently once the growth plates close.
Regulatory status
| Region | Year | Approved for |
|---|---|---|
| USFDA | 2021 | To increase linear growth in paediatric patients with achondroplasia with open epiphyses (accelerated approval) |
| EUEuropean Medicines Agency | 2021 | Treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed |
Status by country
| Jurisdiction | Status | Source |
|---|---|---|
| USUnited States | Authorised medicine | Source 1 |
| EUEuropean Union | Authorised medicine | Source 2 |
| UKUnited Kingdom | No authorised medicine | Source 8 |
| AUAustralia | Authorised medicine | Source 9 |
| CACanada | Authorised medicine | Source 10 |
Trials of Vosoritide to watch
Vosoritide in idiopathic short stature(opens the source in a new tab)
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
CANOPY-HCH-3(opens the source in a new tab)
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Vosoritide in infant hypochondroplasia(opens the source in a new tab)
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Timeline
2028
2026
2021
- Approved, US (FDA)To increase linear growth in paediatric patients with achondroplasia with open epiphyses (accelerated approval)
- Approved, EU (European Medicines Agency)Treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed
Sources
- 01VOXZOGO (vosoritide) for injection, for subcutaneous use: US prescribing information(opens the source in a new tab)
- 02Voxzogo: European public assessment report(opens the source in a new tab)
- 03Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial(opens the source in a new tab)
- 04A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia(opens the source in a new tab)
- 05US Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO (vosoritide) for Children With Achondroplasia(opens the source in a new tab)
- 06BioMarin Announces Positive Results of Phase 3 VOXZOGO (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence(opens the source in a new tab)
- 07C-type natriuretic peptide (NPPC), UniProt P23582(opens the source in a new tab)
- 08MHRA Products: active substances beginning with V(opens the source in a new tab)
- 09VOXZOGO vosoritide 0.4 mg powder for injection vial and diluent pre-filled syringe (376616)(opens the source in a new tab)
- 10Drug Product Database: Voxzogo (DIN 02564661)(opens the source in a new tab)
Educational information, not medical advice. Talk to a licensed clinician before starting, stopping or changing any medicine.