Intractable Rare Dis Res. 2023;12(4):257-261. (DOI: 10.5582/irdr.2023.01055)

Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update

Simran, Devi SSK, Chetan Dushantrao S, Joga R, Kumar S


Dwarfism is a rare condition characterized by small stature. Achondroplasia is predominantly considered the leading cause of dwarfism. Although the condition is not life-threatening, it dramatically impacts the social life of the patient. The United States Food and Drug Administration (US FDA) first approved the drug Voxzogo (vosoritide) for achondroplasia. The drug also received approval from the European Medicines Agency (EMA) via the centralized procedure. The drug is associated with a decrease in blood pressure, a severe adverse event. However, this adverse event/risk has been overcome by benefits, i.e. fulfilling of unmet medical need. In the United States, the drug received accelerated approval as it satisfied the criteria of rare pediatric disease. This review includes a detailed orphan drug approval process with particular reference to vosoritide, which is considered a milestone for the treatment of achondroplasia.

KEYWORDS: US FDA's Orphan Products Grants Program, European Medicines Agency, vosoritide, achondroplasia, rare disease

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