BioCryst Pharmaceuticals, a small drug developer in Durham, said Monday it has been granted Fast Track designation for its experimental drug to fight a rare and potentially fatal genetic disorder called hereditary angioedema.
The Fast Track designation is seen as a vote of confidence from the U.S. Food and Drug Administration, the agency that decides whether drugs are safe and effective for public use. The federal program expedites development of promising drug candidates for treating serious or life-threatening conditions, and will allow BioCryst officials regular feedback from FDA officials as the drug is developed and reviewed.
The BioCryst drug, called BCX4161, will next be tested on nearly 100 people in the United States and Europe in a clinical trial with placebos. The results of this study, expected by the end of this year, will determine how BioCryst and the FDA proceed with further development, said Robert Bennett, BioCryst’s Vice President of Investor Relations & Operations.
Hereditary angioedema, or HAE, afflicts about 1 in 50,000 people with painful swelling of hands, feet, face and airways. Patients experience excruciating pain, nausea, vomiting and, in some instances, asphyxiation.
The primary treatment for HAE is Cinryze, a plasma-derived biologic drug that’s taken through intravenous injection every three to four days, Bennett said.
BCX4161 is made from chemically manufactured molecules and administered as a pill three times a day. BioCryst also has a pill in the development pipeline that could be taken once a day. Both Cinryze and BCX4161 are prophylactic drugs taken to prevent an attack of HAE, rather than to stop an attack in progress.
Shareholders reacted favorably, pushing up BioCryst’s stock by 34 cents, or 3 percent, to close at $11.33 Monday. The stock is down 2 percent over the past year.
Just last month, the FDA approved BioCryst’s first drug, peramivir, an IV drug to treat serious cases of the flu. The federal government spent $235 million on developing the drug, sold under the brand name Rapivab.
BioCryst is also developing BCX4430, an antiviral for the treatment of Ebola virus disease, with $24.4 million in federal funding.
BioCryst was founded in 1986 and went public in 1994. The company employs 49 people.