Approved & therapeutic
Vosoritide
Voxzogo
Vosoritide is a modified analogue of C-type natriuretic peptide approved to increase linear growth in children with achondroplasia who have open epiphyses. It is the first drug directed at the underlying signalling defect rather than at complications.
Also known as: BMN 111, modified CNP
- Status
- Vosoritide is approved by the U.S. FDA and has official prescribing information.
- Evidence
- Approved labeling. Official prescribing information exists, based on clinical trials in people.
- Half-life
- Approximately 25 minutes; the short half-life reflects rapid clearance and is offset by daily dosing.
- How it's taken
- Daily subcutaneous injection
- Mechanism
- Other
How Vosoritide works
Achondroplasia is caused by a gain-of-function mutation in FGFR3, which over-activates the MAPK pathway and suppresses chondrocyte proliferation at the growth plate. Vosoritide binds natriuretic peptide receptor B, and the resulting cGMP signalling inhibits MAPK downstream of FGFR3, relieving that suppression. The molecule is engineered for resistance to neutral endopeptidase, giving it a usable half-life compared with native CNP.
What research has looked at
- The pivotal placebo-controlled trial showed an increase in annualised growth velocity over 52 weeks in children with achondroplasia.
- It addresses the FGFR3 signalling defect itself rather than managing complications.
- Approval requires open epiphyses — the drug acts at the growth plate, so it has no expected effect once growth plates have fused.
Evidence level: Approved labeling. Official prescribing information exists, based on clinical trials in people. These are areas studied or reported, not promised effects.
Reported side effects and risks
- Decreases in blood pressure and associated symptoms including dizziness, fatigue and nausea; the label advises adequate food and fluid intake before dosing and caution in children with significant cardiac or vascular disease.
- Injection-site reactions are very common, including erythema, swelling, pruritus and pain.
- Vomiting and arthralgia.
- Effects on long-term functional outcomes, and on complications of achondroplasia such as spinal stenosis and foramen magnum compression, have not been established — the demonstrated benefit is growth velocity.
Sources
- FDA approved-drug listing for vosoritide · U.S. Food & Drug Administration
- DailyMed prescribing information for vosoritide · NIH · DailyMed
- PubMed research on vosoritide achondroplasia FGFR3 · NIH · National Library of Medicine
- DrugBank record for vosoritide · DrugBank
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Vosoritide FAQ
What is Vosoritide?
Vosoritide is a modified analogue of C-type natriuretic peptide approved to increase linear growth in children with achondroplasia who have open epiphyses. It is the first drug directed at the underlying signalling defect rather than at complications.
How does Vosoritide work?
Achondroplasia is caused by a gain-of-function mutation in FGFR3, which over-activates the MAPK pathway and suppresses chondrocyte proliferation at the growth plate. Vosoritide binds natriuretic peptide receptor B, and the resulting cGMP signalling inhibits MAPK downstream of FGFR3, relieving that suppression. The molecule is engineered for resistance to neutral endopeptidase, giving it a usable half-life compared with native CNP.
Is Vosoritide FDA approved?
Yes. Vosoritide is approved by the U.S. FDA and has official prescribing information. Evidence level: approved labeling. Official prescribing information exists, based on clinical trials in people.
What is the half-life of Vosoritide?
Reported half-life: Approximately 25 minutes; the short half-life reflects rapid clearance and is offset by daily dosing.
How is Vosoritide taken?
Daily subcutaneous injection.
What side effects have been reported with Vosoritide?
Reported in the sources: Decreases in blood pressure and associated symptoms including dizziness, fatigue and nausea; the label advises adequate food and fluid intake before dosing and caution in children with significant cardiac or vascular disease; Injection-site reactions are very common, including erythema, swelling, pruritus and pain; Vomiting and arthralgia; Effects on long-term functional outcomes, and on complications of achondroplasia such as spinal stenosis and foramen magnum compression, have not been established — the demonstrated benefit is growth velocity.
Educational summary compiled from the sources listed above. Not medical advice. Peptide Vault does not sell, source or recommend any compound. Talk to a qualified clinician before starting or changing anything.