Data on burden of HAE, and effectiveness of berotralstat, in children unveiled at 2025 ACAAI Annual Scientific Meeting

A series of scientific presentations demonstrating the impact of HAE on the lives of pediatric patients and caregivers, and the effectiveness of berotralstat (also referred to by its brand name, Orladeyo), have been presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) Orlando, USA, 6-10 November 2025.

Here’s a summary of some of the key findings of the research:

Impact of HAE on pediatric patients and caregivers

  • Adolescents and caregivers report that HAE negatively impacts young patients’ mental health, communication with others, and participation in school, sports, and social activities.
  • 90% of HCPs reported that HAE attacks distress caregivers and patients, disrupt caregiver and patient daily routines, and negatively impact patients’ quality of life.
  • Caregivers reported that attacks occur about every 3 months and rated HAE as moderately severe (mean of 4.0 on a scale of 1-7).
  • Over three-quarters of adolescents (11/14) and nearly half of caregivers (11/23) reported going to the ED or hospital at least once before age 12. Visits ranged from a few hours to a few days in length.
  • Most adolescents and caregivers have experienced negative ED/hospital experiences, including treatment delays, medication unavailability, challenges with medication administration, HCPs unfamiliar with HAE, and heightened stress due to the ED/hospital environment

Effectiveness of berotralstat as a treatment for young people with HAE (2-11 years old)

  • Berotralstat demonstrated early and sustained attack rate reduction, with 65.5% of patients attack-free at month 1 and 70.4% of patients attack-free at month 12. Nearly all (93.1%) patients completed ≥48 weeks of treatment.
  • Berotralstat was safe and well tolerated, with no new safety signals identified. The most commonly reported treatment-emergent adverse event (TEAE) was nasopharyngitis (the common cold).

Dr Donald S. Fong, Chief Medical Officer of BioCryst, said: “Our psychosocial data elucidate that HAE attacks typically begin well before puberty and can have a profoundly negative impact on the mental health and quality of life of both children and their caregivers. Currently there are only injectable treatments approved for prevention of attacks in patients under 12. Berotralstat would be the first and only targeted oral prophylactic therapy for patients with HAE aged 2 to less than 12 years, if approved.”

A New Drug Application (NDA) for an oral granule formulation of berotralstat to prevent HAE attacks in pediatric patients with HAE aged 2 to <12 years is currently under review with the US Food and Drug Administration (FDA), with a target action date of December 12, 2025.

(Source: BioCryst)