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MHLW in Japan granted approval for Vozxogo for injection for the treatment of children with achondroplasia, whose growth plates are not closed.

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Published:22nd Jun 2022

BioMarin Pharmaceutical Inc. announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the registration of Vozxogo (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.

The MHLW in Japan based its decision on the outcomes of a global Phase III randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of Voxzogo and the long-term extension of this Phase III study as well as data from patients participating in a Phase II 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.

Condition: Achondroplasia
Type: drug

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