The FDA has granted Priority Review to BridgeBio’s NDA for oral infigratinib in pediatric achondroplasia, setting a regulatory clock that gives the agency six months rather than the standard ten to reach a decision. For families managing a condition long treated with daily injections, an oral once-daily option would change the daily routine in a concrete way, and BridgeBio already holds Breakthrough Therapy Designation for this program, granted in September 2024.
The NDA rests primarily on PROPEL 3, a Phase 3 trial in 114 children aged 3 to 17 with genetically confirmed achondroplasia and open growth plates. Participants were randomized 2:1, with 75 receiving once-daily oral infigratinib at 0.25 mg/kg and the remainder on placebo. Results published in the New England Journal of Medicine showed a 2.1 cm per year gain in annualized height velocity over placebo. That magnitude, in a population where skeletal growth is the central clinical concern, was enough to anchor both the Breakthrough designation and now the priority filing.
The competitive context matters here. Vosoritide (Voxzogo), a daily subcutaneous injection approved by the FDA in November 2021 for children five and older with achondroplasia, is the current approved standard. Infigratinib targets FGFR3 by a different mechanism and, critically, delivers that effect in a pill rather than an injection. Whether the FDA’s six-month clock produces a 2026 action date or spills into early 2027 depends on when the filing was accepted, which the 8-K does not specify, though the acceptance itself was announced October 6, 2026.
The number to watch is not the PDUFA date, which BridgeBio has not yet disclosed publicly, but the label age range the FDA ultimately accepts. PROPEL 3 enrolled children as young as three, and any restriction below that floor would narrow the addressable population at launch relative to what the trial data actually support.
Source link: https://www.sec.gov/Archives/edgar/data/1743881/000114036126039334/ef20083591_form8k.htm
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
