Our Programs

Pipeline

Our development programs represent the potential to improve the well-being of people whose lives are currently limited by rare disease.

BioCryst Pharmaceuticals discovers novel, oral small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. BioCryst’s core development programs include:

  • BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases
  • BCX9250, an oral ALK-2 inhibitor for treatment of Fibrodysplasia Ossificans Progressiva (FOP)
  • Galidesivir, a broad-spectrum antiviral for serious viral illnesses

These compounds are investigational and have not been deemed safe and effective by the FDA.

BioCryst’s discovery team is also designing drug candidates against additional undisclosed rare disease targets.

For inquiries about licensing opportunities, please contact our business development group.

Program
  • Pre-clinical
  • Phase 1
  • Phase 2
  • Phase 3
  • NDA
  • Approved
Phase
Hereditary Angioedema (HAE)
ORLADEYO® (berotralstat)
Oral Capsule (Prophylactic HAE)
U.S.
Approved
EU + UK
Approved
Japan
Approved
Complement-Mediated Diseases
fibrodysplasia ossificans progressiva (FOP)
infectious diseases

BCX9930, BCX9250 and galidesivir are investigational and have not been deemed safe and effective by the FDA.