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FDA Approved Growth Hormone Axis

Vosoritide

also known as: Voxzogo, BMN 111

The first drug approved for achondroplasia — a CNP analog that stimulates endochondral bone growth by counteracting overactive FGFR3 signaling.

A 39-amino-acid modified CNP analog that activates NPR-B receptors on growth plate chondrocytes to counteract FGFR3-mediated growth inhibition, FDA-approved as a daily subcutaneous injection for achondroplasia in children with open growth plates.

Mechanism of action

Mimics endogenous CNP by binding NPR-B on growth plate chondrocytes. NPR-B activation generates cGMP, which inhibits the overactive FGFR3-MAPK signaling that suppresses bone growth in achondroplasia.

Primary uses

  • Achondroplasia in children with open growth plates (FDA-approved)

Typical dosing

15 mcg/kg once daily (subcutaneous)

15 mcg/kg SC daily. Monitor blood pressure initially.

Regulatory status

FDA-approved November 2021 as Voxzogo for achondroplasia in pediatric patients >=5 years with open epiphyses. Developed by BioMarin.

References

  1. [pubmed] Savarirayan R, et al. "Once-daily vosoritide in children with achondroplasia." Lancet. 2020;396(10252):684-692.

Related peptides

Navepegritide

The weekly achondroplasia treatment — a PEGylated CNP prodrug approved in 2026 that provides continuous growth plate stimulation with once-weekly dosing.

CNP

The third natriuretic peptide — a vascular and skeletal growth factor whose analog vosoritide (Voxzogo) is FDA-approved for achondroplasia, and the endogenous cartilage growth signal.

Somatropin (HGH)

FDA-approved recombinant human growth hormone — the direct hormone replacement, with multiple clinical indications, prescription-only status, and specific federal criminal provisions against off-label distribution.

Lonapegsomatropin

Ascendis Pharma's once-weekly growth hormone prodrug (Skytrofa) — the first weekly GH to receive FDA approval (August 2021) for pediatric GH deficiency in patients aged 1+ years weighing at least 11.5 kg.

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Disclaimer

This entry is for educational purposes only and does not constitute medical advice. Dosing information reflects published regulatory or research data and is not a recommendation. Many compounds described here are not approved for human use in the United States. Consult a licensed medical professional before considering any peptide therapy.