Announcing the financial results for the full year 2019, BioCryst Pharmaceuticals, Inc. President and CEO Jon Stonehouse says that “2020 is off to a strong start, with NDAs accepted and approvals lined up later this year in the U.S. and Japan”:

“We are hearing increasing excitement from both HAE patients and physicians about the availability of an oral option to manage their disease, and we are attracting outstanding commercial talent to bring this new medicine to patients,” Stonehouse added.

Upcoming Key Milestones for the HAE Program – Berotralstat (BCX7353):

  • Submit Marketing Authorization Application (MAA) for oral, once-daily berotralstat for the prevention of HAE attacks with the European Medicines Administration (EMA) (Q1 2020)
  • Approval and launch of oral, once-daily berotralstat in Japan (2H 2020)
  • Approval and launch of oral, once-daily berotralstat in U.S. (December 3, 2020 PDUFA date)

Recent Corporate Developments for the HAE Program – Berotralstat (BCX7353):

  • On February 27, 2020, the company announced it will present abstracts with new data on oral, once-daily berotralstat (BCX7353) at the upcoming annual meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI) March 13-16 in Philadelphia.
  • On February 18, 2020, the company announced that the U.S. Food and Drug Administration (FDA) had accepted and filed its new drug application (NDA) for the approval of oral, once-daily berotralstat (BCX7353) for the prevention of HAE attacks. The Prescription Drug User Fee Act (PDUFA) date for the NDA is December 3, 2020. In the NDA filing acceptance letter, the FDA stated that they are not currently planning to hold an advisory committee meeting to discuss the NDA.
  • On February 3, 2020, the company announced that it had submitted a new drug application (JNDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for approval of oral, once-daily berotralstat for the prophylactic treatment of HAE.
  • On January 12, 2020, the company announced that the APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks, and berotralstat was safe and generally well-tolerated.
  • On December 11, 2019, the company announced it had submitted a new drug application to the FDA for approval of oral, once-daily berotralstat (BCX7353) for the prevention of HAE attacks.

(Source: BioCryst)