Betsy Goodfellow | September 7, 2023 | News story | Research and Development | BridgeBio Pharma, EMA, FDA, Musculo-skeletal disorder, achondroplasia, clinical trial
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BridgeBio Pharma has announced positive feedback from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) on key elements of trial design for its PROPEL3 phase 3 trial of infigratinib in paediatric patients with achondroplasia.
Both the FDA and EMA indicated that a one-year, 2:1 randomised, placebo-controlled trial would be accepted as a registrational study for approval, with the company now planning initiation of the trial by the end of 2023.
The trial intends to assess the safety and efficacy of infigratinib in children between the ages of three and 18 with achondroplasia with open growth plates. The primary endpoint will be change from baseline in AHV, with secondary endpoints including proportionality, height Z-score and impact on medical complications.
Dr Melita Irving, MD, clinical geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, and investigator for the infigratinib clinical programme at the Evelina London Children’s Hospital, commented: “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.”
Adora Ndu, PharmD JD, chief regulatory officer at BridgeBio, added: “We are thrilled to have regulatory alignment on key elements of our phase 3 trial design and the path towards submitting a marketing application to both the FDA and the EMA. We are appreciative of the support from both agencies and plan to move forward swiftly with initiation and dosing in the pivotal trial, with the hopes of providing a much-needed oral option to children with achondroplasia.”
Betsy Goodfellow