Our Programs


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:

  • BCX7353, an oral inhibitor of plasma kallikrein for prevention and treatment of hereditary angioedema (HAE)
  • 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)
  • BCX4430 (galidesivir), a broad-spectrum antiviral for hemorrhagic fevers

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

In December 2014, RAPIVAB® (peramivir injection) was approved by the FDA, the first FDA approval of a drug discovered by BioCryst.

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

  • Pre-clinical
  • Phase 1
  • Phase 2
  • Phase 3
  • Approved
Hereditary Angioedema (HAE)
Complement-Mediated Diseases
BCX9930 – Oral Factor D Inhibitor
Phase 1
fibrodysplasia ossificans progressiva (FOP)
infectious diseases

Targeting Hereditary Angioedema (HAE)

We are working to revolutionize HAE treatment with new and novel orally-administered medicines.

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