BioCryst Launches ORLADEYO® (berotralstat) in Portugal
BioCryst Pharmaceuticals (BCRX) has received a recommendation from Infarmed in Portugal for ORLADEYO® (berotralstat), marking its entry into the Portuguese market. The drug is approved for routine prevention of recurrent hereditary angioedema (HAE) attacks in patients aged 12 and older.
ORLADEYO is significant as the first oral, once-daily therapy for HAE attack reduction in Portugal. This follows the European Commission's marketing authorization granted in April 2021. The drug is now licensed in 44 countries globally, expanding BioCryst's international market presence.
BioCryst Pharmaceuticals (BCRX) ha ricevuto una raccomandazione da Infarmed in Portogallo per ORLADEYO® (berotralstat), segnando il suo ingresso nel mercato portoghese. Il farmaco è approvato per la prevenzione routinaria degli attacchi ricorrenti di angioedema ereditario (HAE) in pazienti di età pari o superiore a 12 anni.
ORLADEYO è significativo in quanto rappresenta la prima terapia orale, da assumere una volta al giorno, per la riduzione degli attacchi di HAE in Portogallo. Questo segue l'autorizzazione alla commercializzazione concessa dalla Commissione Europea nell'aprile 2021. Il farmaco è ora autorizzato in 44 paesi a livello globale, ampliando la presenza internazionale di BioCryst.
BioCryst Pharmaceuticals (BCRX) ha recibido una recomendación de Infarmed en Portugal para ORLADEYO® (berotralstat), marcando su entrada en el mercado portugués. El medicamento está aprobado para la prevención rutinaria de ataques recurrentes de angioedema hereditario (HAE) en pacientes de 12 años o más.
ORLADEYO es significativo ya que es la primera terapia oral, de una vez al día, para la reducción de ataques de HAE en Portugal. Esto sigue a la autorización de comercialización otorgada por la Comisión Europea en abril de 2021. El medicamento ahora está licenciado en 44 países a nivel mundial, ampliando la presencia internacional de BioCryst.
BioCryst Pharmaceuticals (BCRX)는 포르투갈의 Infarmed로부터 ORLADEYO® (berotralstat)에 대한 추천을 받았으며, 이는 포르투갈 시장에 진입하는 것을 의미합니다. 이 약물은 12세 이상의 환자에서 반복적인 유전성 혈관부종(HAE) 발작의 일상적인 예방을 위해 승인되었습니다.
ORLADEYO는 포르투갈에서 HAE 발작 감소를 위한 첫 번째 경구용, 하루 한 번 복용하는 치료제로서 중요합니다. 이는 2021년 4월 유럽연합 집행위원회에서 부여한 마케팅 승인에 따른 것입니다. 이 약물은 현재 전 세계 44개국에서 허가되어 BioCryst의 국제 시장 존재감을 확장하고 있습니다.
BioCryst Pharmaceuticals (BCRX) a reçu une recommandation d'Infarmed au Portugal pour ORLADEYO® (berotralstat), marquant son entrée sur le marché portugais. Le médicament est approuvé pour la prévention routinière des attaques récurrentes d'angioœdème héréditaire (HAE) chez les patients âgés de 12 ans et plus.
ORLADEYO est significatif car il s'agit de la première thérapie orale, à prendre une fois par jour, pour la réduction des attaques d'HAE au Portugal. Cela fait suite à l'autorisation de mise sur le marché accordée par la Commission européenne en avril 2021. Le médicament est désormais autorisé dans 44 pays à travers le monde, élargissant la présence internationale de BioCryst.
BioCryst Pharmaceuticals (BCRX) hat eine Empfehlung von Infarmed in Portugal für ORLADEYO® (berotralstat) erhalten, was den Eintritt in den portugiesischen Markt markiert. Das Medikament ist zur routinemäßigen Prävention von wiederkehrenden hereditären Angioödemen (HAE) bei Patienten ab 12 Jahren zugelassen.
ORLADEYO ist bedeutend, da es die erste orale Therapie ist, die einmal täglich zur Reduzierung von HAE-Attacken in Portugal eingenommen wird. Dies folgt der Marktzulassung, die der Europäischen Kommission im April 2021 erteilt wurde. Das Medikament ist nun in 44 Ländern weltweit lizenziert und erweitert die internationale Marktpräsenz von BioCryst.
- Expansion into Portuguese market with Infarmed recommendation
- First-in-class oral, once-daily therapy for HAE in Portugal
- Achieved licensing in 44 countries globally
- None.
Insights
The Portuguese regulatory approval for ORLADEYO represents a strategic expansion in BioCryst's European commercialization efforts, particularly significant as it includes a recommendation for routine prevention coverage. The Portuguese rare disease market, while smaller compared to major European markets, is notable for several reasons:
The reimbursement recommendation from Infarmed is particularly valuable as it typically leads to consistent coverage through Portugal's National Health Service (SNS). In the context of rare diseases like HAE, which affects approximately 1 in 50,000 people, this translates to a potential patient pool of roughly 200-220 HAE patients in Portugal, considering the country's population of about 10.3 million.
ORLADEYO's oral administration offers a significant competitive advantage over traditional injectable prophylactic treatments. This differentiator typically commands premium pricing in European markets, with annual treatment costs for HAE prophylaxis ranging from
The approval in Portugal brings ORLADEYO's global reach to 44 countries, demonstrating strong execution of BioCryst's international expansion strategy. This systematic approach to market access in Europe strengthens the company's revenue diversification beyond its primary U.S. market. The Portuguese launch could serve as a template for future expansions into other similar-sized European markets, potentially accelerating the drug's adoption across the continent.
RESEARCH TRIANGLE PARK, N.C., Feb. 12, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Infarmed in Portugal has recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older. With this recommendation, HAE patients in Portugal will have access to the first oral, once-daily therapy for the reduction of recurrent HAE attacks.
“The positive Infarmed recommendation of ORLADEYO broadens access to modern prophylaxis, providing greater choice for prescribing physicians and potentially a better quality of life for HAE patients in Portugal,” said Charlie Gayer, chief commercial officer of BioCryst.
The Infarmed decision in Portugal follows the European Commission marketing authorization of ORLADEYO in April 2021. To date, ORLADEYO is licensed in 44 countries.
About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema 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 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 (e.g., 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 (e.g., digoxin) when co-administering 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.
Please see full Prescribing Information.
Please see Portuguese Summary of Product Characteristics and Patient Information Leaflet for ORLADEYO (berotralstat).
SUSPECTED ADVERSE REACTIONS, or side effects, can be reported to Infarmed, the Portuguese National Authority of Medicines and Health Products, preferably by completing the online submission form.
Side effects can also be reported directly to BioCryst Pharmaceuticals at medinfoeurope@biocryst.com or +353 1 699 4405.
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 BioCryst’s plans and expectations for ORLADEYO. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s 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; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance and demand; the FDA, Infarmed, or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; and risks related to the international expansion of BioCryst’s business. 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 actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements.
BCRXW
Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com
Niamh Lyons
+353 87 639 7083
nlyons@biocryst.com
FAQ
What is the significance of ORLADEYO's approval in Portugal for BCRX?
How many countries has BCRX's ORLADEYO been licensed in as of February 2024?
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What age group is ORLADEYO approved for in Portugal?
