Rare Disease

Vosoritide: FDA approved medicine for Achondroplasia

  • May 16, 2023
  • 2 mins read

Voxzogo was approved by the FDA in 2021 to increase linear growth in pediatric patients with open epiphyses with Achondroplasia who are 5 years of age and older. Voxzogo contains Vosoritide, a C-type natriuretic peptide (CNP) analog. This medicine comes in 0.4 mg, 0.56 mg, or 1.2 mg lyophilized powder in a single-dose vial for subcutaneous infusion.

Dosage and Administration
The recommended Voxzogo dosage is based on the patient’s actual body weight. One should administer Voxzogo 0.4 mg by subcutaneous injection once daily.
One should inject Vosoritide at approximately the same time each day, if possible. The volume of Vosoritide to be administered is based on the patient’s actual body weight and the concentration of reconstituted Vosoritide (0.8 mg/mL or 2 mg/mL). Vosoritide must be reconstituted before use.

Warnings And Precautions
Healthcare professionals observed transient decreases in blood pressure in clinical studies of Vosoritide. To decrease the risk of a decrease in blood pressure and associated symptoms (dizziness, fatigue or nausea), instruct patients to be well hydrated and have sufficient food intake before administration of Vosoritide.

Mechanism of Action
In achondroplasia patients, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 (FGFR3). The binding of vosoritide to natriuretic peptide receptor-B (NPR-B) opposes FGFR3 downstream signalling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase. As a result, vosoritide acts as a positive regulator of endochondral bone growth as it promotes chondrocyte proliferation and differentiation.

Vosoritide: Availability in India
Voxzogo (Vosoritide) is not available in India, but multiple licensed pharmaceutical organizations could import this medicine for needy patients in India and other countries. Voxzogo injection cost is reasonable, and one can access this medicine through Indian pharmaceutical companies like Ikris Pharma Network  Biomarin Pharmaceutical is the manufacturer of the medicine.