“This is a transformational year for BioCryst Pharmaceuticals, Inc. as we prepare to launch berotralstat in multiple territories to bring our oral, once-daily prophylactic medicine to HAE patients, and begin generating significant revenue,” says Jon Stonehouse, President and CEO at the announcement of BioCryst’s financial results for the first quarter ended 31 March 2020.
Program Updates and Key Milestones for the HAE Program – Berotralstat (BCX7353): Oral, once-daily treatment for prevention of HAE attacks
- BioCryst expects three regulatory approvals for berotralstat in 2020 and early 2021. These timelines remain on track.
- The U.S. Food and Drug Administration (FDA) is currently reviewing a new drug application for berotralstat and has set an action date of 3 December 2020, under the Prescription Drug User Fee Act (PDUFA).
- In Japan, the Pharmaceuticals and Medical Devices Agency (PMDA) is reviewing a new drug application (JNDA) for berotralstat under the Sakigake timeline, and the company expects approval in Japan in the second half of 2020.
- On 30 March 2020, the company announced that the European Medicines Agency (EMA) had validated its marketing authorization application (MAA) submission for berotralstat and begun their formal review of the MAA under the centralized procedure. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected within approximately 12 months from MAA validation.
- Ongoing commercial launch preparations are on track in the U.S., EU and Japan. The company does not expect delays due to COVID-19.
- On 5 May 2020, the company announced that the United States Patent and Trademark Office issued a notice of allowance for a new composition of matter patent which extends patent protection for berotralstat in the U.S. market by four years through October 2039.