BridgeBio Pharma

New Capabilities

Lead developer of oral achondroplasia treatment

For the estimated 250,000 people worldwide living with achondroplasia—the most common genetic cause of dwarfism—the only approved treatment required daily injections until BioMarin's Voxzogo. On February 12, 2026, BridgeBio reported that its oral pill achieved a 2.10 cm/year increase in height growth over placebo in a Phase 3 trial, exceeding Voxzogo's 1.57 cm/year gain and marking the first statistically significant improvement in body proportionality; two weeks later on February 27, the FDA approved Ascendis Pharma's once-weekly YUVIWEL (navepegritide) for children aged 2 and older.

Updated Mar 1