BioMarin Pharmaceutical Gets Positive European Opinion on Pediatric Drug

By Matt Grossman

BioMarin Pharmaceuticals Inc. has received a positive opinion from the European Committee for Medicines for Human Use for a medicine for the treatment of children with growth disorders.

The drug, vosoritide, is intended to treat achondroplasia, the most common form of disproportionate short stature. Vosoritide is a daily injection for children from 2 years until the end of puberty.

In addition to the positive recommendation, the French National Agency for the Safety of Medicines and Health Products has granted vosoritide a temporary use authorization allowing its use to begin immediately under an authorized process.

Vosoritide would be marketed under the brand name Voxzogo. The European Commission is expected to rule on the drug’s final approval in the third quarter of 2021, according to San Rafael, California-based BioMarin.

Write to Matt Grossman at

Comments are closed.