FDA extends review period for BioCryst pediatric drug application to December

The U.S. Food and Drug Administration has extended its review period for BioCryst Pharmaceuticals’ (NASDAQ:BCRX) new drug application for once-daily ORLADEYO (berotralstat) oral granules intended for pediatric patients with hereditary angioedema aged 2 to 11 years. The company reported Monday that the FDA determined additional final reports and formulation data submitted by BioCryst represented a major amendment to the application, which resulted in a standard three-month extension of the Prescription Drug User Fee Act (PDUFA) goal date.

The new target action date for the FDA’s decision is December 12, 2025. The original review timeline was extended to allow the agency sufficient time to complete a full evaluation of the new information provided.

This information is based on a statement released by BioCryst Pharmaceuticals in a filing with the Securities and Exchange Commission.

In other recent news, BioCryst Pharmaceuticals has reported significant developments regarding its hereditary angioedema (HAE) treatment, ORLADEYO (berotralstat). The company shared data from multiple studies highlighting the long-term efficacy and safety of ORLADEYO across various age groups. Notably, the pediatric APeX-P study showed an 86% reduction in HAE attacks requiring professional care after 12 weeks of treatment. Additionally, BioCryst announced that Colombia’s National Institute of Drug and Food Surveillance has approved ORLADEYO for preventing HAE attacks in patients aged 12 and older, marking its sixth regulatory authorization in Latin America. In Europe, the Netherlands has approved ORLADEYO for reimbursement, expanding its availability across major European countries. Meanwhile, BioCryst has extended its consulting agreement with former Chief Financial Officer Anthony Doyle to ensure continuity in its financial operations. The company remains focused on improving the lives of individuals with rare diseases through its targeted therapeutics.

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