CSL Receives Approval in Japan for ANDEMBRY® (garadacimab) Subcutaneous (S.C.) Injection 200mg Pens, a Novel Human Anti-Activated Factor XII Monoclonal Antibody for the Prevention of Acute Attacks of Hereditary Angioedema (HAE)
CSL Behring has received approval from Japan's MHLW for ANDEMBRY® (garadacimab), a first-in-class monoclonal antibody treatment for preventing hereditary angioedema (HAE) attacks. The treatment, administered via pre-filled pen for once-monthly subcutaneous injection, works by inhibiting activated Factor XII, the initiating factor in the HAE pathway.
The approval follows successful results from the international Phase 3 VANGUARD trial, with the detailed results published in The Lancet (April 2023) and Allergy (October 2024). ANDEMBRY has already received approvals in Australia, the United Kingdom, and the European Union.
HAE is a rare genetic disorder affecting approximately 430 diagnosed patients in Japan, with an estimated total patient population of 2,500 based on global prevalence rates of 1 in 50,000 people. The condition causes recurrent and unpredictable attacks of angioedema in multiple body sites.
CSL Behring ha ricevuto l'approvazione dal MHLW giapponese per ANDEMBRY® (garadacimab), un trattamento monoclonale di prima classe per prevenire gli attacchi di angioedema ereditario (HAE). Il trattamento, somministrato tramite una penna pre-riempita per un'iniezione sottocutanea mensile, agisce inibendo il Fattore XII attivato, il fattore iniziale nella via dell'HAE.
L'approvazione segue risultati positivi dal trial internazionale di Fase 3 VANGUARD, con i risultati dettagliati pubblicati su The Lancet (aprile 2023) e Allergy (ottobre 2024). ANDEMBRY ha già ricevuto approvazioni in Australia, Regno Unito e Unione Europea.
L'HAE è un raro disturbo genetico che colpisce circa 430 pazienti diagnosticati in Giappone, con una popolazione totale stimata di 2.500 pazienti basata su tassi di prevalenza globali di 1 ogni 50.000 persone. La condizione provoca attacchi ricorrenti e imprevedibili di angioedema in più parti del corpo.
CSL Behring ha recibido la aprobación del MHLW de Japón para ANDEMBRY® (garadacimab), un tratamiento de anticuerpo monoclonal de primera clase para prevenir ataques de angioedema hereditario (HAE). El tratamiento, administrado a través de un bolígrafo precargado para una inyección subcutánea mensual, actúa inhibiendo el Factor XII activado, el factor iniciador en la vía del HAE.
La aprobación sigue a resultados exitosos del ensayo internacional de Fase 3 VANGUARD, con los resultados detallados publicados en The Lancet (abril de 2023) y Allergy (octubre de 2024). ANDEMBRY ya ha recibido aprobaciones en Australia, Reino Unido y la Unión Europea.
El HAE es un trastorno genético raro que afecta a aproximadamente 430 pacientes diagnosticados en Japón, con una población total estimada de 2.500 pacientes basada en tasas de prevalencia global de 1 en 50.000 personas. La condición causa ataques recurrentes e impredecibles de angioedema en múltiples partes del cuerpo.
CSL Behring는 일본 MHLW로부터 ANDEMBRY® (가라다시맙)의 승인을 받았습니다. 이는 유전성 혈관 부종(HAE) 공격을 예방하기 위한 최초의 단일클론 항체 치료제입니다. 이 치료는 사전 충전된 펜을 통해 한 달에 한 번 피하 주사로 투여되며, HAE 경로의 시작 인자인 활성화된 제12 응고인자를 억제하여 작용합니다.
이 승인은 국제 3상 VANGUARD 시험의 성공적인 결과에 따른 것으로, 자세한 결과는 The Lancet(2023년 4월)과 Allergy(2024년 10월)에 발표되었습니다. ANDEMBRY는 이미 호주, 영국 및 유럽 연합에서 승인을 받았습니다.
HAE는 일본에서 약 430명의 진단된 환자에게 영향을 미치는 희귀 유전 질환으로, 전 세계 유병률 비율인 5만 명당 1명의 비율을 기준으로 총 환자 수는 약 2,500명으로 추정됩니다. 이 질환은 여러 신체 부위에서 반복적이고 예측할 수 없는 혈관부종 공격을 유발합니다.
CSL Behring a reçu l'approbation du MHLW japonais pour ANDEMBRY® (garadacimab), un traitement par anticorps monoclonal de première classe pour prévenir les attaques d'angioedème héréditaire (HAE). Le traitement, administré par un stylo prérempli pour une injection sous-cutanée mensuelle, agit en inhibant le facteur XII activé, le facteur initiateur dans la voie de l'HAE.
Cette approbation fait suite à des résultats positifs de l'essai international de Phase 3 VANGUARD, dont les résultats détaillés ont été publiés dans The Lancet (avril 2023) et Allergy (octobre 2024). ANDEMBRY a déjà reçu des approbations en Australie, au Royaume-Uni et dans l'Union Européenne.
Le HAE est un trouble génétique rare touchant environ 430 patients diagnostiqués au Japon, avec une population totale estimée à 2 500 patients basée sur des taux de prévalence mondiaux de 1 sur 50 000 personnes. Cette condition provoque des attaques récurrentes et imprévisibles d'angioédème dans plusieurs parties du corps.
CSL Behring hat die Genehmigung des MHLW in Japan für ANDEMBRY® (Garadacimab) erhalten, ein erstklassiger monoklonaler Antikörper zur Prävention von Attacken des hereditären Angioödems (HAE). Die Behandlung, die über einen vorgefüllten Stift für eine monatliche subkutane Injektion verabreicht wird, wirkt, indem sie den aktivierten Faktor XII hemmt, den Initiierungsfaktor im HAE-Weg.
Die Genehmigung folgt auf erfolgreiche Ergebnisse aus der internationalen Phase-3-Studie VANGUARD, deren detaillierte Ergebnisse in The Lancet (April 2023) und Allergy (Oktober 2024) veröffentlicht wurden. ANDEMBRY hat bereits Genehmigungen in Australien, dem Vereinigten Königreich und der Europäischen Union erhalten.
HAE ist eine seltene genetische Erkrankung, die etwa 430 diagnostizierte Patienten in Japan betrifft, mit einer geschätzten Gesamtzahl von 2.500 Patienten basierend auf globalen Prävalenzraten von 1 zu 50.000 Menschen. Die Erkrankung verursacht wiederkehrende und unvorhersehbare Angioödem-Attacken an mehreren Körperstellen.
- First-in-class monoclonal antibody treatment approved for HAE prevention
- Novel once-monthly subcutaneous administration via pre-filled pen
- Multiple regulatory approvals across major markets (Japan, EU, UK, Australia)
- Addresses an underserved market with 2,500 potential patients in Japan
- None.
- ANDEMBRY® is a first-in-class monoclonal antibody treatment that inhibits activated Factor XII (FXIIa), the initiating factor in the HAE pathway, and offers the first pre-filled pen presentation enabling once-monthly subcutaneous administration
- The approval is based on the results of the international pivotal Phase 3 VANGUARD trial, which included HAE patients from
Japan - CSL is dedicated to improving the lives of those with HAE – a community that we have proudly supported for more than 40 years
ANDEMBRY is the first fully human monoclonal antibody in
"ANDEMBRY represents a major advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term disease control through a patient-centric and convenient administration method," said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. "As CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, ANDEMBRY underscores our more than 40-year commitment to HAE research and treatment optimization. This milestone is the result of decades of dedication, and we extend our gratitude to the colleagues, physicians and patients who made this possible 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 are often painful and can occur in multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE is designated as one of
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). A plain language summary of the VANGUARD trial findings has also been published to facilitate understanding of patients and caregivers of the clinical trial data. This summary is accessible in multiple languages, including English and Japanese.
"ANDEMBRY is a breakthrough therapy as the first and only treatment targeting activated Factor XII, the key initiator of HAE attacks," said Dr. Rose Fida, Executive Director and Regional Lead, CSL R&D Japan &
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
About "ANDEMBRY® S.C. Injection 200mg Pens"
Trade name | ANDEMBRY® S.C. Injection 200mg Pens |
Indications or effects | Prevention of acute attacks of Hereditary Angioedema (HAE) |
Dosage and administration | In general, administer subcutaneously the initial loading dose 400 mg of Garadacimab (Genetical Recombination), followed by 200 mg once a month for adults and pediatric patients aged 12 years and older. |
Date of approval | February 20, 2025 |
Manufacturing and marketing | CSL Behring K.K. |
About CSL Behring K.K.
CSL Behring is a global leader in developing and delivering high-quality medicines that treat people with rare and serious diseases. In
For more information, please visit https://www.cslbehring.co.jp.
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.
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FAQ
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