On December 12, 2025, BioCryst Pharmaceuticals Inc (BCRX) announced the U.S. Food and Drug Administration (FDA) approved ORLADEYO (berotralstat) oral pellets for pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years. The 8-K filing disclosed that this approval makes ORLADEYO the first and only targeted oral prophylactic therapy for patients with HAE aged 2 and above, expanding its label from the capsule formulation approved for patients 12 years and older in December 2020.

The approval was supported by interim data from the APeX-P clinical trial, described in the filing as the largest trial to date evaluating a long-term prophylactic therapy for HAE in patients 2 to <12 years of age. According to the filing, ORLADEYO showed early and sustained reductions in monthly attack rates in pediatric HAE patients, with the most commonly reported treatment-emergent adverse event being nasopharyngitis.

BioCryst Pharmaceuticals Inc Chief Executive Officer Jon Stonehouse commented on the significance of the pediatric approval.

"ORLADEYO now first and only targeted oral prophylactic therapy for patients with HAE aged 2 and above." — Jon Stonehouse, Chief Executive Officer, BioCryst Pharmaceuticals Inc

Dr. Raffi Tachdjian, Associate Clinical Professor of Medicine & Pediatrics at the University of California Los Angeles, highlighted the formulation's benefits for younger patients.

"This new oral pellet formulation of ORLADEYO provides a more easily administered long-term-prophylactic option to help children with HAE better manage their disease." — Dr. Raffi Tachdjian, Associate Clinical Professor of Medicine & Pediatrics, University of California Los Angeles

The filing also disclosed that BioCryst Pharmaceuticals Inc has submitted regulatory filings for ORLADEYO oral pellets with the European Medicines Agency and Japan Pharmaceutical and Medical Devices Agency. The company noted that the use of prophylactic therapy has been limited among younger children compared to adults, in part because of the lack of appropriate, less burdensome treatment options.

Charlie Gayer, President & Chief Commercial Officer of BioCryst Pharmaceuticals Inc, commented on the potential impact.

"This approval was supported by positive interim data from the APeX-P clinical trial, the largest trial to date evaluating a long-term prophylactic therapy for HAE in patients 2 to <12 years of age." — Charlie Gayer, President & Chief Commercial Officer, BioCryst Pharmaceuticals Inc

The 8-K filing included several risk factors, noting that other applicable regulatory agencies may not approve ORLADEYO for use in pediatric patients with HAE aged 2 to <12 years within the timeframe expected, or at all. The filing also stated that interim results of a clinical trial do not necessarily predict final results.