A pipeline for complement-mediated and other rare diseases
We are committed to discovering and developing first-in-class or best-in-class oral small-molecule and protein therapeutics that not only have the potential to move the needle, but move mountains, for patients—restoring a sense of freedom for them and their loved ones.
ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
ORLADEYO is a plasma kallikrein inhibitor being investigated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to <12 years of age.
BCX10013 is a potential once-daily, oral Factor D inhibitor for the treatment of complement-mediated diseases.
BCX17725 is a potential KLK-5 inhibitor protein therapeutic for the treatment of Netherton syndrome.
Avoralstat is a potential plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME)
BioCryst’s discovery team is designing oral drug candidates that inhibit the C5 protein, which drives downstream terminal functions.
BioCryst’s discovery team is designing oral drug candidates that inhibit the classical and lectin pathways of the complement system including downstream terminal functions.
BioCryst’s discovery team is designing protein therapeutic candidates that can simultaneously address multiple pathways in the complement system.
*ORLADEYO for pediatric patients, BCX10013, BCX17725, and avoralstat are investigational and have not been deemed safe and effective by the FDA.
†Proof of Concept is typically Phase 1 or 2.
‡Pivotal is typically Phase 3.
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Medicines that meet the needs of patients
For someone living with a complement-mediated or other rare disease, the concept of being able to go about life without a constant reminder of their condition would represent a world of difference. Restoring a new sense of freedom for patients and their loved ones is our goal. This is why we blend cutting-edge technology and complex science to develop first-in-class or best-in-class oral small-molecule and protein therapeutics. Learn more >>