The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing approval for BioCryst Pharmaceuticals, Inc.’s oral, once-daily Orladeyo (berotralstat) 150 mg for prophylactic treatment of HAE in adults and pediatric patients 12 years and older.
Orladeyo is the first and only prophylactic HAE medication approved in Japan. One capsule of Orladeyo per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
Orladeyo will be commercialized in Japan by BioCryst’s partner, Torii Pharmaceutical Co., Ltd. OrphanPacific, Inc. is BioCryst’s representative partner in Japan and holds the marketing authorization.
Torii will launch Orladeyo in Japan following the successful completion of BioCryst’s pricing negotiations with the Japanese National Health Insurance System (NHI).
“Until now, HAE patients in Japan had no therapies approved to prevent attacks, so the approval of Orladeyo marks a significant advance in HAE treatment,” says Goichi Matsuda, President of Torii. “We are pleased to have the opportunity to bring the first oral treatment option to Japanese HAE patients and are actively preparing for the commercialization.”
“The approval of Orladeyo in Japan represents important progress towards our goal to bring an oral, once-daily treatment to HAE patients around the world,” says Jon Stonehouse, President and CEO of BioCryst. “Thank you to the HAE patients who participated in our APeX-J trial, to the investigators who conducted it, and to Torii and OrphanPacific for their partnership to achieve this milestone to offer a much-needed new treatment option to HAE patients and physicians in Japan.”
BioCryst received Orphan Drug and Sakigake designation for Orladeyo in Japan and the approval is based on data from the APeX-J and APeX-2 clinical trials. The APeX-J trial in Japan met its primary endpoint (p=0.003) of a reduction in HAE attacks from baseline for Orladeyo 150 mg compared to placebo, and Orladeyo was safe and generally well-tolerated in the trial. In APeX-2, Orladeyo also met its primary endpoint (p<0.001) for Orladeyo 150 mg compared to placebo and was safe and generally well-tolerated.
In December 2020, the U.S. Food & Drug Administration (FDA) approved Orladeyo in the U.S. In Europe, the European Medicines Agency (EMA) validated its marketing authorization application (MAA) submission for Orladeyo and formal review of the MAA under the centralized procedure is underway. The company expects an approval decision in Europe in the second quarter of 2021.