Shares of BioMarin Pharmaceutical (NASDAQ:BMRN) and Ascendis Pharma (NASDAQ:ASND) fell roughly 6% after rival BridgeBio Pharma (NASDAQ:BBIO) reported encouraging Phase 3 results for its experimental achondroplasia therapy.
BridgeBio said its oral drug infigratinib achieved the primary endpoint in the late-stage PROPEL 3 trial, delivering superior annualized height velocity compared with placebo. The company cited a mean treatment difference of +2.10 cm per year, and noted the therapy also produced the first statistically significant improvement in body proportionality versus placebo in a randomized achondroplasia study.
The update is viewed as a potential competitive challenge to BioMarin’s Voxzogo, currently the only treatment for achondroplasia approved by the U.S. Food and Drug Administration.
Ascendis, meanwhile, is awaiting a regulatory decision on its once-weekly injectable therapy for achondroplasia, with potential FDA approval expected by February 28. Its shares also retreated following the BridgeBio announcement.
Investors appear to be weighing the potential advantages of BridgeBio’s oral formulation, which showed strong efficacy and no serious adverse events linked to the study drug. The company plans to file regulatory submissions in the second half of 2026.
Achondroplasia is a rare genetic disorder that affects bone growth, occurring in roughly one in 15,000 to 40,000 births. An estimated 55,000 people across the U.S. and European Union are living with the condition.