Vosoritide
Vosoritide (BMN 111 / Voxzogo)
First FDA-approved drug for achondroplasia
Vosoritide (Voxzogo) is a modified C-type natriuretic peptide (CNP) analog approved by the FDA for achondroplasia, the most common form of dwarfism. It is the first drug to directly promote bone growth in this condition.
Admin routes
Subcutaneous
Popularity
Niche
Side effects
Generally mild
Vendors
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✓Key benefits
📈What to expect
Measurable increase in growth velocity compared to baseline
Approximately 1.57 cm additional growth versus untreated
Sustained growth improvement maintained through ongoing treatment
Based on community reports and published research. Individual results vary significantly.
💊Dosing protocols
Achondroplasia (approved dose)
15 mcg/kg
Once daily (subcutaneous)
Ongoing until growth plates close
Dosing information is sourced from published research and community protocols. This is not a recommendation. Consult a healthcare professional.
Research status|FDA approved (2021) for achondroplasia in children
Overview
Vosoritide was developed by BioMarin Pharmaceutical and approved by the FDA in November 2021. It is an analog of C-type natriuretic peptide (CNP), a natural hormone that promotes endochondral bone growth. In achondroplasia, a mutation in the FGFR3 gene constitutively activates a signalling cascade that suppresses bone growth at the growth plate. CNP naturally counteracts FGFR3 signalling, but its half-life is only 2-3 minutes. Vosoritide was engineered with an extended half-life, allowing once-daily dosing. In clinical trials, children with achondroplasia treated with vosoritide grew an additional 1.57 cm/year compared to controls.
⚙️How it works
Vosoritide binds to natriuretic peptide receptor B (NPR-B) on chondrocytes in the growth plate. NPR-B activation increases intracellular cGMP, which inhibits the MAPK/ERK signalling pathway. This directly counteracts the overactive FGFR3 signalling that characterises achondroplasia. The result is restored chondrocyte proliferation and differentiation at the growth plate, allowing more normal bone elongation.
⚡Side effects
📅Research history
Phase 2 trial begins in children with achondroplasia
Phase 3 results published in the New England Journal of Medicine
FDA approves vosoritide (Voxzogo) for achondroplasia
A milestone for rare disease treatment
Before vosoritide, there was no pharmacological treatment for achondroplasia. The only options were surgical limb lengthening (painful, complex, with high complication rates) or acceptance. Vosoritide represents a shift from treating symptoms to addressing the molecular cause. The Phase 3 trial showed sustained growth velocity improvement over 5 years of treatment with an acceptable safety profile. It is administered by parents at home via daily subcutaneous injection.
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Core Supplies
Bacteriostatic Water 30mL
Storage & Transport
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Further Reading
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References
- [1]Savarirayan R, et al. 'C-Type natriuretic peptide analogue therapy in children with achondroplasia.' New England Journal of Medicine, 2019.
- [2]BioMarin Pharmaceutical. 'Voxzogo (vosoritide) prescribing information.' 2021.
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Disclaimer: This guide is for educational and informational purposes only. It is not medical advice. The dosing protocols listed are sourced from published research and community reports and do not constitute a recommendation. Always consult a qualified healthcare professional before using any peptide. Check your local regulations regarding peptide purchase and use.