Is vosoritide on the market?

Voxzogo (vosoritide) is a drug used to treat achondroplasia, a genetic disorder that causes severe short stature and uneven development. Vosoritide is an analog of C-type natriuretic peptide (CNP) with proline-glycine at its N-terminus to impart resistance to neutral endopeptidases. It was approved for use under the trade name Voxzogo in the EU in August 2021 and in the United States in November 2021, becoming the first pharmaceutical intervention approved for the treatment of achondroplasia in these two regions. Its manufacturer is BioMarin Pharmaceutical Inc.

The approval from the U.S. Food and Drug Administration (FDA) is based on the results of a global randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo. Voxzogo received accelerated approval based on improved annual growth rate. At Week 52 of the clinical study, the annualized growth rate (AGV) change from baseline was -0.17 cm/year for placebo-treated patients and 1.40 cm/year for Voxzogo-treated patients, resulting in a statistically significant improvement in AGV of 1.57 cm/year in favor of Voxzogo.

Achondroplasia is caused by a genetic mutation that increases the activity of a specific growth regulatory gene called fibroblast growth factor receptor3 (FGFR3). Too much of the protein encoded by the FGFR3 gene can prevent normal bone growth. Vosoritide works by binding to a specific receptor called natriuretic peptide receptor B, which reduces the activity of growth-regulating genes, thereby promoting bone growth. The most common side effects include injection site reactions (such as swelling, redness, itching, or pain), vomiting, and a drop in blood pressure.