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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.
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.