achondroplasia
Stories
-
CHMP adopts positive opinion on Yuviwel for achondroplasia
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion on 15 October 2026, recommending marketing authorisation for Yuviwel (navepegritide) for children 2 years and older with achondroplasia. The opinion notes that Yuviwel improves annualised growth velocity by 1.49 cm versus placebo in a 52-week trial.
-
BioMarin presents new VOXZOGO data at ASBMR 2026
BioMarin will present new data from 13 studies, including a randomized, double-blind, placebo-controlled trial on VOXZOGO's effect on foramen magnum and craniocervical junction growth in children with achondroplasia. The company will also share data on the investigational long-acting medicine BMN 333 and on VOXZOGO's potential use in hypochondroplasia, for which it has submitted a supplemental New Drug Application.