BioCryst Pharmaceuticals has received fast-track designation from the US Food and Drug Administration (FDA) for its BCX4161, an orally administered and selective inhibitor of plasma kallikrein in advanced clinical development to treat hereditary angioedema (HAE).
BioCryst discovered BCX4161, which suppresses bradykinin production by inhibiting plasma kallikrein. Bradykinin is the mediator of acute swelling attacks in HAE patients.
BioCryst Pharmaceuticals president and CEO Jon Stonehouse said: "We are very pleased to have been granted orphan drug and fast-track status from the FDA, as well as recently receiving a positive opinion for orphan drug designation in Europe.
"BCX4161 and our second-generation molecules have the potential to significantly improve HAE patient treatment and their quality of life. We look forward to reporting results from OPuS-2 and sharing updates regarding BCX7353 and our other second-generation HAE assets during 2015."
According to BioCryst, BCX4161 received a positive opinion on the application for orphan drug designation to treat patients with HAE by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).
To demonstrate the efficacy and safety of BCX4161 treatment, BioCryst is enrolling HAE patients in the OPuS-2 trial.
The OPuS-2 trial is a double-blind, randomised and placebo controlled study, which is being conducted for 12 weeks in around 100 patients with HAE, in the US and certain EU countries.
The company plans to report results from the trial by the end of this year.
In addition, the firm is advancing BCX7353 and other second oral kallikrein inhibitors for HAE through preclinical development.
Image: Swollen right hand in a female patient during a hereditary angioedema attack. Photo: courtesy of LucyHAE.