An oral formulation of berotralstat, a potential treatment for children with HAE, to be considered by Federal Drug Administration (FDA) in United States

An announcement regarding a potential new oral granules formula of berotralstat, an HAE medicine which is also called Orladeyo, were provided by the pharmaceutical company BioCryst. The key updates shared with HAEi were:

  • US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for the use of oral, once-daily berotralstat in pediatric patients with hereditary angioedema (HAE) aged 2 to 11 years. The FDA also granted Priority Review of the application, with a Prescription Drug User Fee Act (PDUFA) target action date of 12 September 2025.
  • The NDA was based on positive interim data from the APeX-P clinical trial, the largest trial evaluating a prophylactic therapy for HAE in patients 2 to 11 years of age. Interim results showed berotralstat was well tolerated and demonstrated a very consistent safety profile across this age group, and resulted in early and sustained reductions in monthly attack rates.
  • If approved, berotralstat would be the first targeted oral prophylactic therapy for children with HAE under the age of 12.

Jon Stonehouse, President and Chief Executive Officer of BioCryst, said: “We are excited to take another step closer to bringing berotralstat to younger pediatric patients with HAE. We consistently hear from patients, caregivers and physicians about their desire for a more convenient therapeutic option to treat young children with HAE, and we now may have the opportunity to bring this to them later this year.”

Dr Helen Thackray, Chief Research and Development Officer of BioCryst. “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 HAE 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 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.”

(Source: BioCryst)