BioMarin gets first FDA approval for dwarfism

Nov. 19, 2021

BioMarin Pharmaceutical's once-daily injection for children with the most common type of dwarfism has received clearance from the U.S. FDA, making it the first approved therapy for achondroplasia in the country.

The agency approved Voxzogo, a daily injection, to increase linear growth in achondroplasia patients five years of age and older, who still have open growth plates. The approval was based on the outcomes of a global randomized, double-blind, placebo-controlled phase 3 study evaluating the efficacy and safety of Voxzogo and the open-label extension of the phase 3 study. The study enrolled 121 children aged 5 to 14.

In August 2021, the European Commission also approved Voxzogo. Marketing authorization reviews are in process in JapanBrazil, and Australia with potential approvals in these countries in 2022.

BioMarin expects the therapy's annual per patient revenue in the U.S. to be about $240,000 and anticipates that about 3,000 children with achondroplasia in the country will be eligible for the treatment. BioMarin also won a Rare Pediatric Disease Priority Review Voucher with the approval.

Voxzogo is expected to be available in the U.S. by mid- to late-December.