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BridgeBio Pharma Announces Positive Feedback from the U.S. FDA and EU EMA on the Regulatory Path for a Pivotal Phase 3 Trial of Infigratinib in Children with Achondroplasia
- BridgeBio completed an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), and a scientific advice engagement with the European Union
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- BridgeBio completed an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), and a scientific advice engagement with the European Union (EU) European Medicines Agency (EMA) - FDA and EMA alignment was reached on the adequacy of a one-year, 2:1 randomized, placebo-controlled Phase 3 pivotal trial for infigratinib to support a marketing application for the treatment of children with achondroplasia - The primary endpoint will be change from baseline in annualized height velocity (AHV) at 12 months, with secondary endpoints including proportionality, height Z-score and impact on medical complications - These regulatory interactions clear the path for the initiation of PROPEL3 by the end of 2023 - BridgeBio had previously announced a +3.38cm/yr increase from baseline in annualized height velocity (AHV) at six months at 0.25mg/kg/day, with no serious adverse events (SAEs) or adverse events (AEs) assessed as related to study drug PALO ALTO, Calif., Sept. 06, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive feedback from the U.S. FDA and the EU EMA on key elements of the trial design for PROPEL3, its pivotal Phase 3 study of infigratinib in children with achondroplasia. Both regulatory bodies indicated that a single, one-year, 2:1 randomized, placebo-controlled trial would be acceptable as a registrational study for approval, which clears the path for initiation of the trial by the end of 2023. ”The regulatory feedback clearing the path to approval for infigratinib is great news for children living with achondroplasia and for physicians who treat them. It is incredibly exciting that infigratinib has the potential to become the first effective oral treatment option to improve growth, enhance functionality and decrease complications,” said Dr. Melita Irving, M.D., a clinical geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK and investigator for the infigratinib clinical program at the Evelina London Children’s Hospital. PROPEL3, the Phase 3 registrational study is a randomized, double-blinded, multi-site, placebo-controlled clinical trial, which will evaluate the safety and efficacy of infigratinib in children with achondroplasia aged 3 to 18 years with open growth...