Achondroplasia Pipeline

Nov 21, 2025

Achondroplasia, the most common form of dwarfism, is drawing increasing interest from pharmaceutical companies despite its rarity. Biomarin's VOXZOGO (vosoritide; a C-type natriuretic peptide; once-daily subcutaneous injection) is the only FDA-approved therapy for achondroplasia treatment. When it comes to BioMarin...

Jan 22, 2024

Achondroplasia is a rare genetic disorder affecting bone growth, causing significant short stature, known as dwarfism. This condition is attributed to a fibroblast growth factor receptor 3 (FGFR3) gene mutation. The mutation results in an overactive FGFR3 gene, leading to a slowdown in bone formation within the car...