The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted approval of the registration of BioMarin Pharmaceutical’s Voxzogo (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.
Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.
“We are delighted to offer children in Japan of all ages with achondroplasia access to a treatment option that addresses the underlying genetic mechanism of the condition,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin. “CNP was discovered as a natural regulator of bone growth in Japan in 1990 so we are especially proud to be able to offer a therapeutic choice there. We look forward to nurturing our partnerships with advocates and the achondroplasia community in Japan and beyond.”
The MHLW in Japan based its decision on the outcomes of a global Phase 3 randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of Voxzogo and the long-term extension of this Phase 3 study as well as data from patients participating in a Phase 2 randomized, double-Blind, placebo-controlled clinical trial evaluating the safety and efficacy of Voxzogo in infants and young children with achondroplasia, age 0 to < 60 months.
In 2021, Voxzogo received approvals in the United States, Europe and Brazil.