On December 12, 2025, BioCryst Pharmaceuticals Inc (BCRX) announced FDA approval of ORLADEYO (berotralstat) oral pellets for prophylactic therapy in 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, according to the company's press release attached as Exhibit 99.1.

The FDA approval was supported by positive interim data from the APeX-P clinical trial, described 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.

"ORLADEYO is now the first and only targeted oral prophylactic therapy for patients with HAE aged 2 and above." — BioCryst Pharmaceuticals Inc, 8-K Filing

BioCryst Chief Executive Officer Jon Stonehouse commented on the significance of the approval, stating that ORLADEYO oral pellets have the potential to change how a new generation of children with HAE and their caregivers manage this condition. The company has also filed applications for ORLADEYO oral pellets with the European Medicines Agency and Japan Pharmaceutical and Medical Devices Agency, seeking regulatory approval in additional markets.

Dr. Raffi Tachdjian, Associate Clinical Professor of Medicine & Pediatrics at UCLA's David Geffen School of Medicine, noted that this new oral pellet formulation provides a more easily administered long-term prophylactic option to help children with HAE better manage their disease. The approval expands ORLADEYO's label beyond the capsule formulation approved by the FDA in December 2020 for patients 12 years and older.

"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, UCLA

The filing includes several risk factors, including BioCryst's ability to successfully implement commercialization plans for the ORLADEYO oral pellets and achieve market acceptance. Other risks noted include that interim results of the APeX-P clinical trial do not necessarily predict final results, and that other regulatory agencies may not approve ORLADEYO for pediatric patients within expected timeframes or at all.