Woman in a lab coat and safety glasses operates a laboratory machine, placing a tray of samples into the device.

Pipeline

MAKING “EXTRA- ORDINARY” POSSIBLE STARTS HERE

A pipeline for hereditary angioedema (HAE) and other rare diseases

We are committed to discovering and developing 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.

PhaseAsset	Program	Pre-Clinical	Phase 1	Phase 2	Phase 3/ Pivotal	Approved/ Commercial
Core Programs
ORLADEYO® (berotralstat) Oral Plasma Kallikrein Inhibitor 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.	Hereditary Angioedema (HAE)
ORLADEYO® (berotralstat) Oral Plasma Kallikrein Inhibitor in Pediatrics 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.	Hereditary Angioedema (HAE)
BCX17725 Protein Therapeutic BCX17725 is a KLK5 inhibitor protein therapeutic for the potential treatment of Netherton syndrome.	Netherton Syndrome
Undisclosed BioCryst’s discovery team is focused on rare disease targets where there is significant unmet medical need.	Rare Diseases
Non-Core Programs
RAPIVAB® (peramivir injection) RAPIVAB is an influenza virus neuraminidase inhibitor indicated for the treatment of acute uncomplicated influenza in patients 6 months and older who have been symptomatic for no more than two days.	Infectious Diseases
Avoralstat Ocular Plasma Kallikrein Inhibitor Avoralstat is a plasma kallikrein inhibitor for the potential treatment of diabetic macular edema (DME)	Diabetic Macular Edema (DME)

*ORLADEYO for pediatric patients, BCX17725, and avoralstat are investigational and have not been deemed safe and effective by the FDA.

This page may contain forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results, performance, or achievements to be materially different from any future results or performance expressed or implied on this webpage. You should not place undue reliance on the forward-looking statements. For additional information, including important risk factors, please refer to BioCryst’s documents filed with the SEC and located at https://ir.biocryst.com/financial-information/sec-filings.

Close-up of various labeled laboratory sample vials containing different liquids and substances, arranged on a white paper towel on a lab bench.

Medicines that meet the needs of patients

For someone living with a 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 >>