BioCryst Announces FDA Acceptance of NDA for ORLADEYO® (berotralstat) Oral Granules in Patients with Hereditary Angioedema Aged 2 to 11 Years

About this update from Biocryst Pharmaceuticals, Inc.

–FDA grants Priority Review of application, with PDUFA target action date of September 12 , 2025– – ORLADEYO would be the first targeted oral prophylactic therapy for patients with HAE under the age of 12, if approved – RESEARCH TRIANGLE PARK, N.C. , May 14, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today 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 also granted Priority Review of the application, with a Prescription Drug User Fee Act (PDUFA) target action date of September 12, 2025 . “We are excited to take another step closer to bringing ORLADEYO to younger pediatric patients with HAE. We consistently hear from patients, caregivers and physicians about their desire for a more convenient therapeutic option to treat young children with HAE, and we now may have the opportunity to bring this to them later this year,” said Jon Stonehouse , president and chief executive officer of BioCryst. The NDA was based on positive interim data from the APeX-P clinical trial, the largest trial evaluating a prophylactic therapy for HAE in patients 2 to 11 years of age. Interim results from APeX-P that were presented at the 2025 American Academy of Allergy , Asthma & Immunology / World Allergy Organization Joint Congress earlier this year showed ORLADEYO was well tolerated and demonstrated a very consistent safety profile across this age group, and resulted in early and sustained reductions in monthly attack rates. “As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of HAE symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far. If approved, we believe this oral granule formulation of ORLADEYO could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays,” said Dr. Helen Thackray , chief research and development officer of BioCryst. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE under the age of 12, if approved. BioCryst 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 . Additional regulatory filings are 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. About ORLADEYO ® (berotralstat) ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema ( HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATION ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema ( HAE) in adults and pediatric patients 12 years and older. Limitations of use The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATION An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients

View stock analysis, news, and events for Biocryst Pharmaceuticals, Inc.