Hereditary Angioedema and HAE Management
Biocryst Pharmaceuticals Inc. focuses on clinical research in hereditary angioedema, with attention to long-term treatment use, ongoing safety evaluation, and symptom control in affected patients.
- Berotralstat safety
- Long-term access in HAE
- Oral therapy for HAE attacks
Its research activity includes both adult and pediatric populations, reflecting interest in treatment continuity across different age groups.
Pediatric Hereditary Angioedema
The sponsor funds studies examining HAE in children, with emphasis on how oral treatment behaves in younger patients and how it may be used in routine care.
- Pediatric pharmacokinetics
- Children aged 2 to under 12 years
- Treatment tolerability
This area includes evaluation of oral administration in pediatric subjects weighing at least 12 kg.
Netherton Syndrome
Biocryst Pharmaceuticals Inc. also supports clinical research in Netherton syndrome, a rare dermatologic and systemic disorder, with studies centered on therapeutic potential and treatment safety.
- BCX17725
- Adult and adolescent participants
- Safety and tolerability
Research in this area includes assessment of treatment exposure and serum concentration behavior in affected participants.
Pharmacokinetics and Immunogenicity
A key part of the sponsor’s clinical portfolio involves measuring pharmacokinetic properties and immunogenicity for oral therapies used in HAE and Netherton syndrome.
- Serum concentration profiling
- Oral drug exposure
- Immune response monitoring
These studies support the characterization of treatment behavior in both adult and pediatric populations.
