Biocryst Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has accepted its new drug application (NDA) for the use of oral, once-daily Orladeyo® (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to 11 years. The FDA has also granted priority review of the application, with a Prescription Drug User Fee Act (PDUFA) target action date of September 12, 2025.
The NDA was based on positive interim data from the APEX-P clinical trial, which is the largest trial evaluating a prophylactic therapy for HAE in patients 2 to 11 years of age. Interim results from APEX-P showed that Orladeyo was well-tolerated and demonstrated a very consistent safety profile across this age group, as well as resulting in early and sustained reductions in monthly attack rates.
The company's President and CEO, Jon Stonehouse, expressed excitement about the possibility of bringing Orladeyo to younger pediatric patients with HAE, addressing the desire for a more convenient therapeutic option for treating young children with HAE.
If approved, Orladeyo would be the first targeted oral prophylactic therapy for children with HAE under the age of 12. The company has also filed its line extension application for the use of Orladeyo oral granules in patients with HAE aged 2 to 11 years with the European Medicines Agency, with additional regulatory filings planned in other global territories, including Japan and Canada.
Orladeyo received FDA approval in December 2020 for prophylaxis to prevent HAE attacks in adult and pediatric patients 12 years and older and is now commercially available in more than 30 countries.
The safety and effectiveness of Orladeyo in pediatric patients under 12 years of age have not been established, and there are insufficient data available to inform drug-related risks with Orladeyo use in pregnancy. Furthermore, the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production are not known.
Biocryst Pharmaceuticals is a global biotechnology company with a commitment to improving the lives of people living with hereditary angioedema and other rare diseases. The company has commercialized Orladeyo, the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. Today the company's shares have moved -2.71% to a price of $10.06. If you want to know more, read the company's complete 8-K report here.
