- The stock price of BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) is trading higher than 5% pre-market. This is why it happened.
The stock price of BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) — a biotechnology company that discovers oral and small-molecule medicines — is trading higher than 5% pre-market. Investors are responding to the Ministry of Health, Labor and Welfare (MHLW) in Japan granting marketing and manufacturing approval for oral, once-daily ORLADEYO (berotralstat) 150 mg for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
ORLADEYO is going to be the first and only prophylactic HAE medication approved in Japan. And one capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. And Torii is going to launch ORLADEYO in Japan following the successful completion of BioCryst’s pricing negotiations with the Japanese National Health Insurance System (NHI).
BioCryst had 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. And 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.
Last month, 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 a formal review of the MAA under the centralized procedure is underway. BioCryst expects an approval decision in Europe in the second quarter of 2021.
And with the approval in Japan, BioCryst is eligible to receive an additional milestone payment of $15 million from Torii upon receipt of a reimbursement price approval from Japan’s National Health Insurance system in excess of the threshold specified in the agreement with Torii. Plus BioCryst will receive tiered royalties ranging from 20% to potentially 40% of Japanese net sales.
“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. 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.”
— Goichi Matsuda, president of Torii
“Today’s approval of ORLADEYO in Japan represents important progress towards our goal to bring an oral, once-daily treatment to HAE patients around the world. 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.”
— Jon Stonehouse, president and chief executive officer of BioCryst
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