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vosoritide (Voxzogo)

Indications: - treatment of achondroplasia in children >= 2 years whose epiphyses are not yet closed Dosage: - 15 ug/ug/day SC Adverse effects: - injection site reactions (edema, eythema, itching or pain) - vomiting - hypotension Mechanism of action: - analog of C-type natriuretic peptide

Related

achondroplasia

General

metabolic agent (metabolic modifier)

Database Correlations

PUBCHEM cid=119058036

References

  1. FDA News Release. Nov 19, 2021 FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-improve-growth-children-most-common-form-dwarfism
  2. Wikipedia: Vosoritide https://en.wikipedia.org/wiki/Vosoritide
  3. Savarirayan R, Tofts L, Irving M et al. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med 2021. 23:2443-2447 PMID: 34341520 PMCID: PMC8327889 Free PMC article
  4. Duggan S Vosoritide: First Approval Drugs. 2021 Nov;81(17):2057-2062 PMID: 34694597
  5. Savarirayan R, Tofts L, Irving M et al Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2021. Sept 5, Volume 396, ISSUE 10252, P684-692 https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(20)31541-5/fulltext
  6. HIGHLIGHTS OF PRESCRIBING INFORMATION Voxzogo (vosoritide) for injection, for subcutaneous use https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf