BioCryst Announces FDA Acceptance of NDA for ORLADEYO® (berotralstat) Oral Granules in Patients with Hereditary Angioedema Aged 2 to 11 Years
The FDA has accepted BioCryst Pharmaceuticals' (BCRX) New Drug Application for ORLADEYO® oral granules to treat hereditary angioedema (HAE) in children aged 2-11 years. The FDA granted Priority Review with a PDUFA target date of September 12, 2025.
The application is based on positive interim data from the APeX-P clinical trial, showing ORLADEYO was well-tolerated with sustained reductions in monthly attack rates. The trial revealed HAE symptoms typically onset at age two, indicating a significant early disease burden. If approved, ORLADEYO would become the first targeted oral prophylactic therapy for HAE patients under 12 years old.
BioCryst has also filed for approval in Europe, with plans for additional submissions in Japan and Canada. ORLADEYO is currently approved for patients 12 years and older in over 30 countries since December 2020.
La FDA ha accettato la domanda di nuovo farmaco di BioCryst Pharmaceuticals (BCRX) per le granulazioni orali di ORLADEYO® per il trattamento dell'angioedema ereditario (HAE) nei bambini di età compresa tra 2 e 11 anni. La FDA ha concesso una Revisione Prioritaria con una data target PDUFA fissata per il 12 settembre 2025.
La domanda si basa su dati intermedi positivi dello studio clinico APeX-P, che mostrano come ORLADEYO sia ben tollerato e determini una riduzione costante del tasso mensile di attacchi. Lo studio ha evidenziato che i sintomi dell'HAE di solito insorgono all'età di due anni, indicando un significativo carico precoce della malattia. In caso di approvazione, ORLADEYO diventerebbe la prima terapia profilattica orale mirata per i pazienti con HAE sotto i 12 anni.
BioCryst ha inoltre presentato richiesta di approvazione in Europa, con piani per ulteriori sottomissioni in Giappone e Canada. ORLADEYO è attualmente approvato per pazienti di 12 anni e più in oltre 30 paesi dal dicembre 2020.
La FDA ha aceptado la solicitud de nuevo medicamento de BioCryst Pharmaceuticals (BCRX) para los gránulos orales de ORLADEYO® para tratar el angioedema hereditario (HAE) en niños de 2 a 11 años. La FDA otorgó una Revisión Prioritaria con una fecha objetivo PDUFA para el 12 de septiembre de 2025.
La solicitud se basa en datos interinos positivos del ensayo clínico APeX-P, que muestran que ORLADEYO fue bien tolerado con reducciones sostenidas en las tasas mensuales de ataques. El ensayo reveló que los síntomas del HAE suelen comenzar a los dos años, indicando una carga significativa de la enfermedad desde temprana edad. Si se aprueba, ORLADEYO sería la primera terapia profiláctica oral dirigida para pacientes con HAE menores de 12 años.
BioCryst también ha presentado solicitudes de aprobación en Europa, con planes para más presentaciones en Japón y Canadá. ORLADEYO está aprobado actualmente para pacientes de 12 años en adelante en más de 30 países desde diciembre de 2020.
FDA가 BioCryst Pharmaceuticals(BCRX)의 ORLADEYO® 경구 과립제에 대한 신약 신청서를 승인했습니다. 이 약은 2세에서 11세 사이의 유전성 혈관부종(HAE) 치료에 사용됩니다. FDA는 우선 심사(Priority Review)를 부여했으며, PDUFA 목표일은 2025년 9월 12일입니다.
이 신청서는 APeX-P 임상시험의 긍정적인 중간 데이터에 기반하며, ORLADEYO가 잘 견뎌졌고 월별 발작률이 지속적으로 감소함을 보여줍니다. 임상시험 결과 HAE 증상은 보통 2세에 시작되어 조기 질병 부담이 상당함을 나타냅니다. 승인될 경우 ORLADEYO는 12세 미만 HAE 환자를 위한 최초의 표적 경구 예방 치료제가 됩니다.
BioCryst는 유럽에서도 승인 신청을 했으며, 일본과 캐나다에서도 추가 제출을 계획 중입니다. ORLADEYO는 현재 2020년 12월부터 12세 이상 환자에게 30개국 이상에서 승인받아 사용되고 있습니다.
La FDA a accepté la demande de nouveau médicament de BioCryst Pharmaceuticals (BCRX) pour les granules oraux ORLADEYO® destinés au traitement de l'angio-œdème héréditaire (HAE) chez les enfants âgés de 2 à 11 ans. La FDA a accordé une révision prioritaire avec une date cible PDUFA fixée au 12 septembre 2025.
La demande repose sur des données intermédiaires positives de l'essai clinique APeX-P, montrant qu'ORLADEYO est bien toléré avec une réduction soutenue du taux mensuel d'attaques. L'essai a révélé que les symptômes de l'HAE apparaissent généralement vers l'âge de deux ans, indiquant une charge précoce significative de la maladie. En cas d'approbation, ORLADEYO deviendrait la première thérapie prophylactique orale ciblée pour les patients atteints d'HAE de moins de 12 ans.
BioCryst a également déposé une demande d'approbation en Europe, avec des plans de soumissions supplémentaires au Japon et au Canada. ORLADEYO est actuellement approuvé pour les patients de 12 ans et plus dans plus de 30 pays depuis décembre 2020.
Die FDA hat den Antrag von BioCryst Pharmaceuticals (BCRX) für ORLADEYO® orale Granulate zur Behandlung von hereditärem Angioödem (HAE) bei Kindern im Alter von 2 bis 11 Jahren akzeptiert. Die FDA gewährte eine Prioritätsprüfung mit einem PDUFA-Zieldatum am 12. September 2025.
Der Antrag basiert auf positiven Zwischenergebnissen der APeX-P klinischen Studie, die zeigten, dass ORLADEYO gut verträglich ist und eine anhaltende Reduktion der monatlichen Anfallraten bewirkt. Die Studie ergab, dass HAE-Symptome typischerweise im Alter von zwei Jahren beginnen, was auf eine erhebliche frühe Krankheitsbelastung hinweist. Bei Zulassung wäre ORLADEYO die erste gezielte orale prophylaktische Therapie für HAE-Patienten unter 12 Jahren.
BioCryst hat zudem Zulassungsanträge in Europa eingereicht und plant weitere Einreichungen in Japan und Kanada. ORLADEYO ist derzeit seit Dezember 2020 für Patienten ab 12 Jahren in über 30 Ländern zugelassen.
- FDA granted Priority Review status, potentially expediting the approval process
- Positive interim clinical trial data showing good tolerability and reduced attack rates
- Would be the first oral prophylactic therapy for HAE patients under 12 years old
- Expanding market potential with applications filed in multiple global territories
- None.
Insights
BioCryst's ORLADEYO for pediatric HAE receives FDA Priority Review, potentially becoming first oral prophylactic for children under 12.
The FDA's acceptance of BioCryst's New Drug Application (NDA) for ORLADEYO with Priority Review status represents a significant regulatory milestone. Priority Review designation is reserved for drugs that would provide significant improvements in the safety or effectiveness of treating serious conditions, reducing the standard 10-month review period to 6 months. The September 12, 2025 PDUFA date creates a clear timeline for potential market entry.
The regulatory submission is particularly notable because it addresses a critical unmet need in hereditary angioedema (HAE) treatment for children aged 2-11. The APeX-P trial data revealed that the median age of HAE symptom onset was just two years, indicating the disease burden begins far earlier than previously recognized in many patients. Currently, there are no FDA-approved oral prophylactic treatments specifically for this young population.
If approved, ORLADEYO would gain a significant first-mover advantage in this pediatric segment, complementing its existing approval for patients 12 and older received in December 2020. The company is pursuing a comprehensive global regulatory strategy with simultaneous filings with the European Medicines Agency and planned submissions in Japan and Canada, potentially creating multiple revenue expansion opportunities across key pharmaceutical markets.
The oral granule formulation represents a child-friendly delivery method that could substantially improve treatment adherence compared to injectable alternatives, addressing both medical needs and quality-of-life considerations for young patients and their caregivers.
ORLADEYO's potential approval offers first oral HAE prophylaxis for children 2-11, addressing early disease onset and reducing emergency interventions.
The APeX-P clinical trial findings represent a significant advancement in our understanding of hereditary angioedema in pediatric populations. The trial revealed that HAE symptom onset occurs at a median age of just two years, substantially earlier than previously documented in the medical literature. This data fundamentally shifts our clinical perspective on HAE as a disease with significant pediatric onset that requires earlier intervention strategies.
The interim results demonstrate that ORLADEYO provided early and sustained reductions in monthly attack rates while maintaining a consistent safety profile across the 2-11 age group. This is particularly important because HAE attacks in children can be difficult to diagnose and manage, often leading to unnecessary emergency department visits, hospitalizations, and psychological trauma.
Current prophylactic options for young children with HAE are extremely limited and typically involve complex injectable therapies that create significant treatment burden for both patients and caregivers. The oral granule formulation of ORLADEYO represents a potential paradigm shift in treatment modality, significantly improving the feasibility of consistent prophylaxis in this challenging age group.
From a clinical perspective, effective prophylaxis in early childhood could potentially alter the natural history of HAE by reducing the cumulative burden of attacks during critical developmental periods. By preventing acute HAE episodes that require emergency interventions, this therapy could substantially reduce healthcare utilization while improving quality of life for affected children and their families.
–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 (≥
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 <12 years of age have not been established.
There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.
To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; BioCryst’s ability to successfully progress its development plans as described herein, including meeting the expected timelines; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; the commercial viability of ORLADEYO for use in pediatric patients with HAE aged 2 to 11 years, including its ability to achieve sustained market acceptance; and the FDA, European Medicines Agency or other applicable regulatory agency may not approve ORLADEYO for use in pediatric patients with HAE aged 2 to 11 years within the timeframe expected, or at all, may ultimately determine that there are deficiencies in the development program or execution thereof, may require additional information or studies, may disagree with our safety and efficacy conclusions, may impose certain restrictions, warnings, or other requirements, may impose a clinical hold with respect to ORLADEYO, or may withhold, delay, or withdraw market approval for ORLADEYO. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.
BCRXW
Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com
FAQ
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