BioCryst Pharmaceuticals, Inc. has reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for HAE with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan.
The Phase 3 trial design agreed upon for Japan, APeX-J, is a randomized, placebo-controlled double-blind trial of 24 weeks duration with a 28-week safety extension. Approximately 24 Japanese subjects with HAE will be enrolled. The APeX-J trial design closely follows the design of APeX-2, a Phase 3 clinical trial being conducted in the U.S., Canada, and European countries. APeX-J tests the same dose levels of BCX7353 as in APeX-2, 110 mg daily and 150 mg daily, and the endpoints are identical to those in APeX-2.Data from the APeX-J and APeX-2 trials will be combined for regulatory submission in Japan.
“Japanese HAE patients would welcome an oral prophylactic drug as another treatment alternative, which provides HAE patients ease in daily life as well as relief from travelling outside their hometown for treatment to receive on-demand therapy of HAE attacks in a hospital,” said Professor Michihiro Hide, M.D., Ph.D, Dean of the School of Medicine and Professor and Chairman, Department of Dermatology, Hiroshima University, and a leading expert in HAE in Japan.
BCX7353 was one of the first products granted Sakigake designation by the Japanese PMDA in October 2015. Sakigake designation is awarded to innovative products addressing an unmet medical need in Japan or providing improved efficacy compared to available treatment. Sakigake designation is associated with accelerated review of a Japanese NDA as well as pricing and other corporate benefits for the holder.
“Now that we have agreement on the regulatory requirements for marketing authorization of BCX7353 in Japan, we can move forward with executing APeX-J and selecting a commercialization partner for this region,” said Jon P. Stonehouse, President & CEO. “A once-a-day oral treatment for this rare disease represents a high unmet medical need in Japan and an excellent partnering opportunity.”