The journal Allergy has published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating oral, once-daily berotralstat for the prophylactic treatment of HAE.
The APeX-J trial met its primary endpoint of a reduction in the rate of HAE attacks for berotralstat 150 mg compared to placebo during the 24-week period (p=0.003). Berotralstat was safe and generally well-tolerated in the trial.
These results are consistent with the global phase 3 APeX-2 trial, where berotralstat 150 mg also reduced the rate of HAE attacks compared to placebo (p<0.001) and was safe and generally well-tolerated.
“APeX-J is the first placebo-controlled trial of an HAE medicine conducted in Japan and the berotralstat data are very exciting for patients, who currently have no approved prophylactic treatment options. Based on the safety and efficacy profile, I believe berotralstat, if approved, would be an important advancement in HAE management for Japanese patients,” says Dr. Isao Ohsawa, President of Saiyu Soka hospital and principal investigator of the APeX-J trial.
Dr. Ohsawa and the study authors note that HAE is estimated to affect 2,500 patients in Japan and the recognition of HAE by physicians is low. Although two on-demand treatments are approved, no therapies are currently approved for long-term prophylaxis in Japan.
“Berotralstat would be the first approved prophylactic therapy for HAE patients in Japan and we believe there is a significant opportunity for berotralstat to accelerate the diagnosis of HAE patients and dramatically improve the quality of life for patients,” says Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc.
A new drug application (JNDA) is under review in Japan for approval of oral, once-daily berotralstat for the prophylactic treatment of HAE. Berotralstat is being reviewed under Sakigake designation and the company expects a decision on approval in December 2020.(Source: BioCryst)
