The US Food and Drug Administration (FDA) has accepted BioCryst Pharmaceuticals’ new drug application (NDA) for Orladeyo (berotralstat), granting it priority review for use in children aged two to 11 with hereditary angioedema (HAE).
A Prescription Drug User Fee Act target action date was set by the agency for 12 September 2025.
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The application was supported by the APeX-P trial’s positive interim data. This trial assessed a prophylactic HAE therapy in these subjects.
The results indicated that the therapy was well tolerated and maintained a consistent safety profile in this young age group. It also led to sustained and early minimisations of monthly attack rates.
If approved, this plasma kallikrein inhibitor will be the inaugural oral targeted prophylactic therapy for HAE patients under 12.
Beyond the US, the company has also submitted a line extension application to the European Medicines Agency for the use of the therapy oral granules in this patient population.
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By GlobalDataThe company plans additional regulatory filings in other worldwide territories, including Canada and Japan.
BioCryst Pharmaceuticals chief research and development officer Dr Helen Thackray stated: “As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far.
“If approved, we believe this oral granule formulation of Orladeyo could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays.”
In December 2020, the therapy was approved by the US regulator initially for HAE attack prevention in the adult population and in children aged 12 and above. It is currently available commercially in 30 countries.
in 2024, the company secured a contract worth up to $69m from the US Department of Health and Human Services for the procurement of Rapivab to treat influenza.
