Feb. 12 at 1:34 PM
Shares of BioMarin and Ascendis Pharma declined after BridgeBio reported positive Phase 3 results for its oral achondroplasia drug, infigratinib. In the PROPEL 3 trial, the treatment met its primary endpoint, showing a +2.10 cm/year improvement in annualized height velocity versus placebo, along with the first statistically significant improvement in body proportionality in a randomized study.
The results pose a competitive threat to BioMarin’s FDA-approved Voxzogo and to Ascendis’ weekly injectable therapy, currently under FDA review with a decision expected by Feb. 28. BridgeBio’s oral option may offer a key advantage over injectable treatments and showed no drug-related serious adverse events. Regulatory filings are planned for the second half of 2026.
Achondroplasia affects roughly 1 in 15,000–40,000 births, impacting about 55,000 people across the U.S. and EU.
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