BioCryst Pharmaceuticals, Inc., a small pharmaceutical company headquartered in Durham, North Carolina announced on January 26 that one of the drugs in the company’s pipeline, BCX4161 has been granted fast track status by the FDA.

BCX4161 is a novel and selective inhibitor of plasma kallikrein discovered by BioCryst and is in development for the treatment of hereditary angioedema (HAE). HAE is a rare and hereditary disease affecting approximately 1 in 10,000 to 1 in 50,000 people.  The gene responsible for HAE is called C1-inhibitor (C1NH).  HAE patients suffer episodic attacks of edema in various parts of the body including hands, feet, face and airway.  In addition, patients often have bouts of excruciating abdominal pain and other GI track symptoms due to intestinal edema.  Severe airway edema can lead to death by asphyxiation.  BCX4161 inhibits plasma kallikrein and consequently suppresses bradykinin production. Bradykinin is the mediator of acute swelling attacks in HAE patients. Current treatment options include purified C1 inhibitor concentrate (Cinryze, Berinert, or Ruconest), Ecallantide (a kallibrein inhibitor) and Icatibant (a bradykinin B2 receptor antagonist).  These drugs are in either intravenous or subcutaneous injection form with various adverse effects.  BCX4161 is being developed as an orally administered drug and has the potential to significantly improve HAE patient treatment and their quality of life.

 

Related Links:

http://www.haea.org/patients/what-is-hae/
http://www.omim.org/entry/106100