U.S. FDA Grants Fast Track Designation for BioCryst’s HAE Drug

Rare Daily Staff

The U.S. Food and Drug Administration granted BioCryst Pharmaceuticals Fast Track designation for BCX7353 for the prevention of angioedema attacks in patients with hereditary angioedema, a rare and potentially life-threating condition.

HAE is a rare, genetic disorder that results in recurring attacks of swelling in various parts of the body, including the abdomen, face, feet, genitals, hands and throat that can be can debilitating and painful. Attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.

BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE.

BCX7353 was generally safe and well tolerated in a phase 2 clinical trial. BioCryst is currently conducting the phase 3 study and the long-term safety study, both evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. BioCryst is also conducting a proof-of-concept phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the acute treatment of angioedema attacks.

Fast Track Designation provides opportunities for frequent interactions with the FDA during development of a product candidate and provides the opportunity for priority review if supported by clinical data at the time of the new drug application submission.

“Fast Track Designation from the FDA serves as another indicator of the importance of BCX7353 to meet an unmet medical need for HAE patients in the U.S.,” said Jon Stonehouse, president CEO of BioCryst. “We remain focused on completing the phase 3 program and preparing for an NDA filing in the second half of 2019.”

August 8, 2018
Photo: Jon Stonehouse, president CEO of BioCryst