BridgeBio Pharma’s (Nasdaq: BBIO) shares were up nearly 5% at $30.37 in early trading, after the company announced sustained positive results from PROPEL 2, a Phase II trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating continued potential best-in-class efficacy and an encouraging safety profile.
Infigratinib is an oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia and hypochondroplasia, both forms of dwarfism, at their source.
Under the trade name Truseltiq, infigratinib gained accelerated US regulatory approval in 2021 for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma. Mizhuo Securities analysts recently estimated peak sales for infigratinib of $1.2 billion in achondroplasia. However the US Food and Drug Administration withdrew approval of the drug last month, at the request of BridgeBio, which cited difficulties in recruiting and enrolling study subjects for the required confirmatory clinical trial in first line cholangiocarcinoma.
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