European Commission Approves CSL's ANDEMBRY® (garadacimab) for the Prevention of Recurrent Attacks of Hereditary Angioedema (HAE)
CSL (CSLLY) has received European Commission approval for ANDEMBRY® (garadacimab), the first and only once-monthly treatment targeting factor XIIa for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older. This marks the third regulatory approval for ANDEMBRY, following recent approvals in Australia and the UK.
The treatment comes with a patient-centric pre-filled pen for subcutaneous self-injection and works by inhibiting plasma protein factor XIIa. The approval is based on the Phase 3 VANGUARD trial and its open-label extension study, with results published in The Lancet (April 2023) and Allergy (October 2024).
HAE is a rare genetic disorder affecting approximately 1 in 50,000 people, characterized by recurrent and unpredictable attacks of angioedema. The treatment will be available following access and reimbursement negotiations, and is currently under review in the US, Japan, Switzerland, and Canada.
CSL (CSLLY) ha ricevuto l'approvazione della Commissione Europea per ANDEMBRY® (garadacimab), il primo e unico trattamento mensile che mira al fattore XIIa per prevenire gli attacchi di angioedema ereditario (HAE) in pazienti di età pari o superiore a 12 anni. Questo segna la terza approvazione regolatoria per ANDEMBRY, dopo le recenti approvazioni in Australia e nel Regno Unito.
Il trattamento è fornito con una penna pre-riempita centrata sul paziente per l'auto-iniezione sottocutanea e agisce inibendo la proteina plasmatica fattore XIIa. L'approvazione si basa sullo studio di Fase 3 VANGUARD e sul suo studio di estensione in aperto, con risultati pubblicati su The Lancet (aprile 2023) e Allergy (ottobre 2024).
HAE è un raro disturbo genetico che colpisce circa 1 persona ogni 50.000, caratterizzato da attacchi ricorrenti e imprevedibili di angioedema. Il trattamento sarà disponibile dopo le negoziazioni per l'accesso e il rimborso, ed è attualmente in fase di revisione negli Stati Uniti, Giappone, Svizzera e Canada.
CSL (CSLLY) ha recibido la aprobación de la Comisión Europea para ANDEMBRY® (garadacimab), el primer y único tratamiento mensual que se dirige al factor XIIa para prevenir los ataques de angioedema hereditario (HAE) en pacientes de 12 años o más. Esto marca la tercera aprobación regulatoria para ANDEMBRY, tras las recientes aprobaciones en Australia y el Reino Unido.
El tratamiento se presenta en una pluma precargada centrada en el paciente para la auto-inyección subcutánea y actúa inhibiendo la proteína plasmática factor XIIa. La aprobación se basa en el ensayo de Fase 3 VANGUARD y su estudio de extensión abierto, con resultados publicados en The Lancet (abril de 2023) y Allergy (octubre de 2024).
HAE es un trastorno genético raro que afecta aproximadamente a 1 de cada 50,000 personas, caracterizado por ataques recurrentes e impredecibles de angioedema. El tratamiento estará disponible tras las negociaciones de acceso y reembolso, y actualmente está en revisión en EE. UU., Japón, Suiza y Canadá.
CSL (CSLLY)는 ANDEMBRY® (가라다시맙)에 대해 유럽연합 집행위원회의 승인을 받았습니다. 이 치료는 12세 이상의 환자에서 유전성 혈관부종(HAE) 공격을 예방하기 위해 XIIa 인자를 타겟으로 하는 첫 번째이자 유일한 월 1회 치료법입니다. 이는 호주와 영국에서의 최근 승인에 이어 ANDEMBRY에 대한 세 번째 규제 승인을 의미합니다.
이 치료법은 피하 자가 주사를 위한 환자 중심의 프리필드 펜으로 제공되며, 혈장 단백질 XIIa 인자를 억제하는 방식으로 작용합니다. 승인은 3상 VANGUARD 시험과 그 공개 연장 연구를 기반으로 하며, 결과는 The Lancet(2023년 4월)과 Allergy(2024년 10월)에 발표되었습니다.
HAE는 약 50,000명 중 1명에게 영향을 미치는 드문 유전 질환으로, 반복적이고 예측할 수 없는 혈관부종 공격이 특징입니다. 이 치료법은 접근 및 환급 협상 후에 제공될 예정이며, 현재 미국, 일본, 스위스 및 캐나다에서 검토 중입니다.
CSL (CSLLY) a reçu l'approbation de la Commission européenne pour ANDEMBRY® (garadacimab), le premier et unique traitement mensuel ciblant le facteur XIIa pour prévenir les attaques d'angioedème héréditaire (HAE) chez les patients âgés de 12 ans et plus. Cela marque la troisième approbation réglementaire pour ANDEMBRY, après les récentes approbations en Australie et au Royaume-Uni.
Le traitement est fourni avec un stylo prérempli centré sur le patient pour l'auto-injection sous-cutanée et agit en inhibant la protéine plasmatique facteur XIIa. L'approbation est basée sur l'essai de phase 3 VANGUARD et son étude d'extension en ouvert, dont les résultats ont été publiés dans The Lancet (avril 2023) et Allergy (octobre 2024).
Le HAE est une maladie génétique rare qui touche environ 1 personne sur 50 000, caractérisée par des attaques récurrentes et imprévisibles d'angioedème. Le traitement sera disponible après des négociations d'accès et de remboursement, et est actuellement en cours d'examen aux États-Unis, au Japon, en Suisse et au Canada.
CSL (CSLLY) hat die Genehmigung der Europäischen Kommission für ANDEMBRY® (garadacimab) erhalten, die erste und einzige einmal monatliche Behandlung, die auf Faktor XIIa abzielt, um Angioödeme aufgrund von hereditärem Angioödem (HAE) bei Patienten ab 12 Jahren zu verhindern. Dies ist die dritte behördliche Genehmigung für ANDEMBRY, nach den jüngsten Genehmigungen in Australien und dem Vereinigten Königreich.
Die Behandlung erfolgt mit einem patientenzentrierten, vorgefüllten Pen für die subkutane Selbstinjektion und wirkt, indem sie das Plasmaprotein Faktor XIIa hemmt. Die Genehmigung basiert auf der Phase-3-Studie VANGUARD und deren offener Verlängerungsstudie, deren Ergebnisse in The Lancet (April 2023) und Allergy (Oktober 2024) veröffentlicht wurden.
HAE ist eine seltene genetische Erkrankung, die etwa 1 von 50.000 Personen betrifft und durch wiederkehrende und unvorhersehbare Angioödem-Attacken gekennzeichnet ist. Die Behandlung wird nach Verhandlungen über Zugang und Erstattung verfügbar sein und befindet sich derzeit in der Überprüfung in den USA, Japan, der Schweiz und Kanada.
- First and only once-monthly treatment for HAE prevention
- Received third major regulatory approval (EC, TGA, MHRA)
- Convenient administration through pre-filled pen auto-injector
- Novel mechanism targeting factor XIIa at cascade initiation
- Product availability dependent on pending access and reimbursement negotiations
- market size with HAE affecting only 1 in 50,000 people
- ANDEMBRY®, the first and only once-monthly treatment targeting factor XIIa to prevent attacks in HAE patients, inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body
- ANDEMBRY reinforces CSL's decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection
- Supported by the Phase 3 VANGUARD trial, this marks the third regulatory approval for ANDEMBRY, which was also recently approved by
MARBURG, Germany, Feb. 13, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that the European Commission (EC) has approved ANDEMBRY® (garadacimab), the first and only once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older. ANDEMBRY inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body. ANDEMBRY reinforces CSL's decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection.
"ANDEMBRY is a significant advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method," said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. "ANDEMBRY, CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients. Thank you to all the colleagues, physicians and patients who contributed to this exciting milestone for HAE patients and CSL."
HAE is a rare, chronic, debilitating, and potentially life-threatening genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks of HAE are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE occurs in about 1 in 50,000 people of any ethnic group.
"The physical and emotional toll of HAE is substantial, and the true prevalence of the disease could be higher than currently recorded due to patient misdiagnoses," said Prof. Dr. med Markus Magerl, MD, Head of Clinical Trials, Charité University Hospital Berlin, Institute of Allergology. "The unpredictable nature of HAE is one of the most daunting aspects for many patients, who never know when an attack might occur. Current HAE preventive therapies work at various downstream steps in the cascade, but none prevent the cascade at its very start like ANDEMBRY."
The approval of ANDEMBRY is based on the efficacy and safety data from the pivotal international Phase 3 VANGUARD trial and its open-label extension study. The detailed results of the VANGUARD trial were published in The Lancet in April 2023 and the primary results of the ongoing open-label extension study were published in Allergy (October 2024).
"Garadacimab, a novel once monthly subcutaneous treatment that inhibits activated Factor XII, is a welcome addition to the HAE treatment landscape," said Henrik Balle Boysen, President, HAE International (HAEi). "People with HAE now have another choice for lessening the burden associated with this lifelong condition and realizing the community's shared goal of experiencing life to the fullest."
CSL Behring will make ANDEMBRY available for eligible people upon completion of access and reimbursement negotiations.
The centralized marketing authorization of ANDEMBRY is valid in all European Union (EU) member states as well as in the European Economic Area (EEA) countries
About ANDEMBRY® (garadacimab)
ANDEMBRY (garadacimab) is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) that has completed Phase 3 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. ANDEMBRY is CSL's first homegrown recombinant monoclonal antibody to gain approval. It was discovered and optimized by scientists at CSL's Bio21-based research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility. ANDEMBRY uniquely inhibits the plasma protein, FXIIa. When FXII is activated, it initiates the cascade of events leading to edema formation. By targeting FXIIa, ANDEMBRY inhibits this cascade at the top as compared to other HAE therapies that target downstream mediators.
As of February 2025, ANDEMBRY has been approved by the Australian Therapeutic Goods Administration (TGA) on January 14, 2025, the
Warnings: Do not use ANDEMBRY® if you are allergic to garadacimab, or any of the ingredients listed at the end of this leaflet. Always check the ingredients to make sure you can use this medicine.
Precautions: Patients should inform their doctor if they have any other medical conditions or take any medicines for any other condition. Patients should inform their doctor if they are pregnant or intend to become pregnant or are breastfeeding or intend to breastfeed.
Interactions: Patients should inform their doctor if they are taking any other medicines, including any medicines, vitamins or supplements that you buy without a prescription from your pharmacy, supermarket or health food shop.
Side effects: The most commonly observed side effects associated with ANDEMBRY® in the phase 3 clinical trial were injection site reactions (2/39,
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL.
For more information about CSL, visit www.CSL.com.
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SOURCE CSL Behring
FAQ
What is the significance of ANDEMBRY's EC approval for CSL stock (CSLLY)?
When will ANDEMBRY be available in European markets for CSLLY investors?
What other markets has CSLLY received approval for ANDEMBRY in 2025?
How does ANDEMBRY's mechanism of action differ from existing HAE treatments?
