On December 12, 2025, BioCryst Pharmaceuticals Inc (BCRX) announced FDA approval of 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, 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, which the company 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 during the trial, 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 pediatric approval in the filing, stating that the new oral pellet formulation provides a more easily administered long-term prophylactic option. Dr. Raffi Tachdjian, Associate Clinical Professor of Medicine & Pediatrics at UCLA's David Geffen School of Medicine, also commented on the benefits of the oral pellet formulation for younger patients.

The company has filed applications for ORLADEYO oral pellets with the European Medicines Agency and Japan Pharmaceutical and Medical Devices Agency, according to the December 12, 2025 filing. These regulatory submissions seek approval for the same pediatric indication of prophylactic therapy for patients with hereditary angioedema aged 2 to <12 years.

"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." — Jon Stonehouse, Chief Executive Officer, BioCryst Pharmaceuticals Inc

The filing 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, commented on the potential impact of the oral pellet formulation on children with HAE in the disclosed materials.

BioCryst's 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 company also disclosed risks related to its ability to successfully implement commercialization plans for the ORLADEYO oral pellets and achieve market acceptance.