CSL Receives Positive CHMP Opinion for Garadacimab in Hereditary Angioedema (HAE)
CSL (ASX:CSL; USOTC:CSLLY) received a positive CHMP opinion recommending marketing authorization for garadacimab, a first-of-its-kind once-monthly treatment for hereditary angioedema (HAE) prevention in patients aged 12 and older. The treatment, which targets factor XIIa to prevent HAE attacks, demonstrated impressive results in the Phase 3 VANGUARD trial, with 62% of patients achieving attack-free status and an 86.5% reduction in mean monthly HAE attacks compared to placebo.
The European Commission's final decision is expected in Q1 2025. The recommendation is supported by favorable long-term safety data from an ongoing open-label extension study, with median exposure of 13.8 months. If approved, garadacimab would be available across all EU member states.
CSL (ASX:CSL; USOTC:CSLLY) ha ricevuto un'opinione positiva dal CHMP che raccomanda l'autorizzazione al commercio per garadacimab, un trattamento innovativo da somministrare una volta al mese per la prevenzione dell'angioedema ereditario (HAE) nei pazienti di età pari o superiore a 12 anni. Il trattamento, che agisce sul fattore XIIa per prevenire gli attacchi di HAE, ha mostrato risultati impressionanti nello studio di Fase 3 VANGUARD, con il 62% dei pazienti che ha raggiunto uno stato senza attacchi e una riduzione dell'86,5% nel numero medio di attacchi mensili di HAE rispetto al placebo.
La decisione finale della Commissione Europea è attesa per il primo trimestre del 2025. La raccomandazione è supportata da dati di sicurezza a lungo termine favorevoli provenienti da uno studio di estensione in aperto in corso, con un'esposizione mediana di 13,8 mesi. Se approvato, garadacimab sarà disponibile in tutti gli stati membri dell'UE.
CSL (ASX:CSL; USOTC:CSLLY) recibió una opinión positiva del CHMP recomendando la autorización de comercialización para garadacimab, un tratamiento único administrado una vez al mes para la prevención del angioedema hereditario (HAE) en pacientes de 12 años o más. El tratamiento, que se dirige al factor XIIa para prevenir los ataques de HAE, mostró resultados impresionantes en el ensayo de Fase 3 VANGUARD, con el 62% de los pacientes alcanzando un estado libre de ataques y una reducción del 86,5% en el número medio de ataques mensuales de HAE en comparación con el placebo.
Se espera que la decisión final de la Comisión Europea llegue en el primer trimestre de 2025. La recomendación se apoya en datos de seguridad a largo plazo favorables de un estudio de extensión en abierto en curso, con una exposición mediana de 13,8 meses. Si se aprueba, garadacimab estará disponible en todos los estados miembros de la UE.
CSL (ASX:CSL; USOTC:CSLLY)는 가라다시맙에 대한 마케팅 허가를 추천하는 긍정적인 CHMP 의견을 받았습니다. 이 치료제는 12세 이상의 환자에서 유전성 혈관부종(HAE) 예방을 위한 최초의 월 1회 치료입니다. HAE 발작을 예방하기 위해 XIIa 인자를 목표로 하는 이 치료는 3상 VANGUARD 시험에서 인상적인 결과를 보였으며, 62%의 환자가 발작 없는 상태를 유지했습니다 그리고 평균 월 HAE 발작이 86.5% 감소했습니다 (플라시보와 비교).
유럽연합 집행위원회의 최종 결정은 2025년 1분기에 예상됩니다. 이 추천은 진행 중인 공개 연장 연구에서의 장기 안전성 데이터에 의해 뒷받침되며, 중간 노출 기간은 13.8개월입니다. 승인된다면, 가라다시맙은 모든 EU 회원국에서 이용 가능할 것입니다.
CSL (ASX:CSL; USOTC:CSLLY) a reçu un avis positif du CHMP recommandant l'autorisation de mise sur le marché pour garadacimab, un traitement unique à administration mensuelle pour la prévention de l'angioedème héréditaire (HAE) chez les patients âgés de 12 ans et plus. Le traitement, qui cible le facteur XIIa pour prévenir les attaques de HAE, a démontré des résultats impressionnants dans l'essai de phase 3 VANGUARD, avec 62 % des patients atteignant un statut sans attaque et une réduction de 86,5 % des attaques mensuelles moyennes de HAE par rapport au placebo.
La décision finale de la Commission européenne est attendue au premier trimestre 2025. La recommandation est soutenue par des données de sécurité à long terme favorables provenant d'une étude d'extension à ouverture en cours, avec une exposition médiane de 13,8 mois. Si elle est approuvée, garadacimab sera disponible dans tous les États membres de l'UE.
CSL (ASX:CSL; USOTC:CSLLY) erhielt eine positive CHMP-Empfehlung zur Genehmigung der Vermarktung von garadacimab, einer einzigartigen monatlichen Behandlung zur Prävention von hereditärem Angioödem (HAE) bei Patienten ab 12 Jahren. Die Behandlung, die auf Faktor XIIa abzielt, um HAE-Anfälle zu verhindern, zeigte im Phase-3-VANGUARD-Studie beeindruckende Ergebnisse, wobei 62 % der Patienten einen anfallfreien Status erreichten und eine Reduktion von 86,5 % bei den durchschnittlichen monatlichen HAE-Anfällen im Vergleich zu Placebo festgestellt wurde.
Die endgültige Entscheidung der Europäischen Kommission wird im ersten Quartal 2025 erwartet. Die Empfehlung wird durch langfristige Sicherheitsdaten einer laufenden offenen Erweiterungsstudie unterstützt, die eine mittlere Exposition von 13,8 Monaten aufweist. Bei Genehmigung wäre garadacimab in allen EU-Mitgliedstaaten verfügbar.
- First-in-class once-monthly treatment for HAE prevention
- 62% of patients achieved attack-free status in Phase 3 trial
- 86.5% reduction in mean monthly HAE attacks vs placebo
- Favorable long-term safety profile demonstrated in extension study
- None.
If approved, garadacimab will be the first and only once-monthly treatment inhibiting factor XIIa to prevent attacks in HAE patients – a community CSL has been serving for more than 40 years
MARBURG,
"CSL has a longstanding and relentless patient-focused approach to developing transformational medicines in areas of unmet need. This CHMP decision brings us closer to offering an innovative treatment to patients living with HAE, which is a debilitating and potentially life-threatening condition," said Emmanuelle Lecomte-Brisset, Senior Vice President and Head of Global Regulatory Affairs, CSL. "We look forward to making this therapy available to patients in
Attacks of HAE are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities. Current HAE preventive therapies work at various downstream steps in the cascade, but none prevent the cascade at its start.
The CHMP positive opinion is based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study, both evaluating the efficacy and safety of garadacimab. The pivotal study met its primary endpoint and showed that garadacimab led to 62 percent of patients achieving attack-free status throughout the treatment period, reduced the median number of HAE attacks to zero and reduced the mean number of HAE attacks per month by 86.5 percent compared to placebo. Interim analysis of the ongoing open-label extension study (median garadacimab exposure 13.8 months) showed that garadacimab has a favorable long-term safety profile and provides sustained HAE attack reduction. The full results from VANGUARD were published in The Lancet (April 2023) and the primary results of the ongoing open-label extension study were published in Allergy (October 2024).
If granted, the centralized marketing authorization of garadacimab would be valid in all EU member states. The final opinion was published in the CHMP meeting highlights on the EMA website.
About HAE
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death.
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.
Media Contact
Valerie Bomberger, CSL
Office: +1 610-291-5388
Mobile: +1 267-280-3829
Email: valerie.bomberger@cslbehring.com
Stephanie Fuchs, CSL Behring
Mobile: +49 151 58438860
Email: Stephanie.Fuchs@cslbehring.com
In Australia:
Jimmy Baker, CSL
Mobile: +61 450 909 211
Email: Jimmy.Baker@csl.com.au
Chris Cooper, CSL
Mobile: +61 455 022 740
Email: Chris.cooper@csl.com.au
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SOURCE CSL Behring
FAQ
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