The European Medicines Agency (EMA) has validated the marketing authorization application (MAA) submission for approval of oral, once-daily berotralstat (BCX7353) for the prevention of HAE attacks. With this validation, the EMA has begun their formal review of the MAA under the centralized procedure for all member states of the European Union, Norway, Iceland, and Liechtenstein.
An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected within approximately 12 months.
“Berotralstat would represent the first targeted oral therapy approved for HAE prophylaxis in Europe and would deliver a major advance in therapy to HAE patients. HAE treatment in Europe tends to be consolidated and we have developed excellent relationships with HAE-treating physicians through our clinical trials. This is allowing us to build an efficient and experienced European commercial team to bring our innovative medicine to patients,” says Jon Stonehouse, CEO of BioCryst Pharmaceuticals, Inc.
BioCryst expects three regulatory approvals for berotralstat in 2020 and early 2021. 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 Japanese approval in the second half of 2020.