Daily Medication Pearl: Vosoritide (Voxzogo) for Achondroplasia

Vosoritide is indicated to increase linear growth in pediatric patients with achondroplasia.

Medication Pearl of the Day: Vosoritide (Voxzogo)

Indication: Vosoritide is a C-type natriuretic peptide (CNP) analog indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.

Insight:

  • Dosing: Recommended dosage is based on patient’s weight. Administer subcutaneously once daily.
  • Dosage forms: For injection 0.4 mg, 0.56 mg, or 1.2 mg lyophilized powder in a single-dose vial for reconstitution.
  • Adverse events (AEs): Most common AEs (>10%) are injection site erythema, injection site swelling, vomiting, injection site urticaria, arthralgia, decreased blood pressure, and gastroenteritis.
  • Mechanism of action: Binding of vosoritide to natriuretic peptide receptor-B antagonizes FGFR3 downstream signaling by inhibiting the extracellular signal-regulated kinases 1 and 2 in the mitogen-activated protein kinase pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase. As a result, vosoritide, like CNP, acts as a positive regulator of endochondral bone growth as it promotes chondrocyte proliferation and differentiation.
  • Manufacturer: Biomarin Pharma

Sources:

label (fda.gov)

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