Market reaction
BioMarin Pharmaceutical (NASDAQ:BMRN) and Ascendis Pharma (NASDAQ:ASND) saw their stocks decline by roughly 6% following the release of clinical data from BridgeBio Pharma (NASDAQ:BBIO) on an achondroplasia therapy.
Study results
BridgeBio reported that its oral agent, infigratinib, achieved the primary endpoint in the Phase 3 PROPEL 3 study, producing superior annualized height velocity compared with placebo with a mean treatment difference of +2.10 cm/year. The company also reported that infigratinib produced the first statistically significant improvement in body proportionality against placebo in a randomized achondroplasia trial.
Safety and regulatory plan
The company said that the trial did not identify any serious adverse events related to the study drug. BridgeBio indicated plans to submit regulatory applications in the second half of 2026.
Competitive implications
The results introduce potential competitive pressure for existing therapies. BioMarin’s Voxzogo is identified as the only FDA-approved treatment for achondroplasia at present. Ascendis is developing a once-weekly injection for the condition that is under FDA review and carries a potential approval date of February 28. The market reaction reflected investor reassessment of how an oral option could alter the competitive landscape for achondroplasia treatments.
Condition background as stated by companies
Achondroplasia is described as a genetic condition affecting bone growth. It is estimated to occur in approximately one in every 15,000 to 40,000 newborns and to affect an estimated 55,000 people across the U.S. and the European Union.
Summary and outlook
BridgeBio’s Phase 3 readout for oral infigratinib — showing a +2.10 cm/year mean treatment difference in annualized height velocity, a first statistically significant body proportionality benefit in a randomized trial, and no reported serious adverse events related to the drug — was followed by share-price declines of about 6% for BioMarin and Ascendis. BridgeBio plans regulatory submissions in the second half of 2026. The information presented here reflects the trial results and stated regulatory plans without further interpretation.
Key points
- BridgeBio's oral infigratinib met the primary endpoint in Phase 3 PROPEL 3 with a mean treatment difference of +2.10 cm/year in annualized height velocity versus placebo.
- The trial produced the first statistically significant improvement in body proportionality against placebo in a randomized achondroplasia study.
- BioMarin's Voxzogo is currently the only FDA-approved treatment for achondroplasia; Ascendis has a once-weekly injectable under FDA review with a potential approval by February 28.
Risks and uncertainties
- Regulatory timing and outcomes remain uncertain - BridgeBio plans regulatory submissions in the second half of 2026, and Ascendis’ product is under FDA review with a potential approval by February 28.
- Market dynamics could shift depending on how regulators evaluate efficacy and safety data; reported absence of serious adverse events in the study is limited to the trial's findings.
- Investor reaction to clinical trial readouts can affect biotech and healthcare equity valuations, contributing to volatility in pharmaceutical and biotech stocks.