Astellas Submits New Drug Application for Conditional Approval of Avacincaptad Pegol for Geographic Atrophy in Japan
Astellas Pharma has submitted a New Drug Application (NDA) to Japan's Ministry of Health for avacincaptad pegol (ACP), potentially becoming the first treatment for geographic atrophy (GA) in Japan. GA, affecting over 5 million people globally, is a progressive form of age-related macular degeneration that can cause irreversible vision loss.
The submission is supported by GATHER1 and GATHER2 clinical trials, which demonstrated ACP's ability to slow GA lesion growth with a favorable safety profile over two years. Currently, GA has no approved treatments outside the US and Australia, and an estimated 66% of GA patients may become legally blind or severely visually impaired without timely treatment.
Astellas Pharma ha presentato una Nuova Richiesta di Farmaco (NDA) al Ministero della Salute giapponese per avacincaptad pegol (ACP), divenendo potenzialmente il primo trattamento per l'atrofia geografica (GA) in Giappone. La GA, che colpisce oltre 5 milioni di persone in tutto il mondo, è una forma progressiva di degenerazione maculare legata all'età che può causare perdita della vista irreversibile.
La presentazione è supportata dagli studi clinici GATHER1 e GATHER2, che hanno dimostrato la capacità di ACP di rallentare la crescita delle lesioni della GA con un profilo di sicurezza favorevole nell'arco di due anni. Attualmente, la GA non ha trattamenti approvati al di fuori degli Stati Uniti e dell'Australia, e si stima che il 66% dei pazienti affetti da GA possa diventare legalmente cieco o gravemente ipovedente senza un trattamento tempestivo.
Astellas Pharma ha presentado una Nueva Solicitud de Medicamento (NDA) al Ministerio de Salud de Japón para avacincaptad pegol (ACP), convirtiéndose potencialmente en el primer tratamiento para la atrofia geográfica (GA) en Japón. La GA, que afecta a más de 5 millones de personas en todo el mundo, es una forma progresiva de degeneración macular relacionada con la edad que puede causar pérdida de visión irreversible.
La presentación está respaldada por los ensayos clínicos GATHER1 y GATHER2, que demostraron la capacidad de ACP para ralentizar el crecimiento de las lesiones de GA con un perfil de seguridad favorable durante dos años. Actualmente, la GA no tiene tratamientos aprobados fuera de los Estados Unidos y Australia, y se estima que el 66% de los pacientes con GA pueden volverse legalmente ciegos o tener una discapacidad visual severa sin un tratamiento oportuno.
아스텔라스 제약(Astellas Pharma)는 일본 보건부에 아바신캡타드 페골(avacincaptad pegol, ACP)에 대한 신약 승인 요청서(NDA)를 제출했으며, 이는 일본에서 지리적 위축(GA)에 대한 첫 번째 치료제가 될 가능성이 있습니다. GA는 전 세계적으로 500만 명 이상이 영향을 받는 진행성 노인성 황반 변성의 한 형태로, 회복 불가능한 시력 손실을 초래할 수 있습니다.
이번 제출은 GATHER1 및 GATHER2 임상 시험의 지원을 받았으며, 이 시험들은 ACP가 GA 병변의 성장을 늦추고 2년 동안 우호적인 안전성 프로파일을 유지할 수 있음을 입증했습니다. 현재, GA는 미국과 호주 이외에서는 승인된 치료법이 없으며, GA 환자의 66%가 적절한 치료를 받지 않으면 법적으로 실명하거나 심각한 시각 장애를 겪을 수 있을 것으로 추정됩니다.
Astellas Pharma a soumis une Demande de Médicament Nouveau (NDA) au Ministère de la Santé du Japon pour avacincaptad pegol (ACP), pouvant potentiellement devenir le premier traitement pour l'atrophie géographique (GA) au Japon. La GA, qui affecte plus de 5 millions de personnes dans le monde, est une forme progressive de dégénérescence maculaire liée à l'âge qui peut provoquer une perte de vision irréversible.
La soumission est soutenue par les essais cliniques GATHER1 et GATHER2, qui ont démontré la capacité de l'ACP à ralentir la croissance des lésions de GA avec un profil de sécurité favorable sur deux ans. Actuellement, il n'existe aucun traitement approuvé pour la GA en dehors des États-Unis et de l'Australie, et il est estimé que 66 % des patients atteints de GA pourraient devenir légalement aveugles ou gravement malvoyants sans traitement rapide.
Astellas Pharma hat einen Antrag auf Zulassung eines neuen Arzneimittels (NDA) beim japanischen Gesundheitsministerium für avacincaptad pegol (ACP) eingereicht, wodurch es potenziell die erste Behandlung für die geografische Atrophie (GA) in Japan werden könnte. GA betrifft weltweit über 5 Millionen Menschen und ist eine fortschreitende Form der altersbedingten Makuladegeneration, die zu irreversiblen Sehverlust führen kann.
Die Einreichung wird durch die klinischen Studien GATHER1 und GATHER2 unterstützt, die gezeigt haben, dass ACP das Wachstum von GA-Läsionen verlangsamen kann und über zwei Jahre hinweg ein günstiges Sicherheitsprofil aufweist. Derzeit gibt es außerhalb der USA und Australiens keine zugelassenen Behandlungen für GA, und schätzungsweise 66 % der GA-Patienten könnten ohne rechtzeitige Behandlung rechtlich blind oder schwer sehbehindert werden.
- Potential first-mover advantage in Japanese market for GA treatment
- Strong clinical trial results showing efficacy in slowing disease progression
- Large addressable market with over 5 million GA patients globally
- Demonstrated sustained efficacy over two years in clinical trials
- No immediate financial impact expected for fiscal year ending March 2025
- geographical approval (currently only US and Australia)
- Ministry of Health, Labour and Welfare to evaluate ACP as potential first and only treatment for patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in
GA is a progressive form of AMD that can cause irreversible vision loss, with no treatments currently approved outside the US or
Marci English, Vice President, Head of BioPharma and Ophthalmology Development, Astellas
"Today's submission comes as good news to people in
The NDA submission is based on results of overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled clinical trials, which evaluated the safety and efficacy of monthly 2mg intravitreal administration of ACP in patients with GA secondary to AMD.6 The data from both trials demonstrates that ACP slows GA lesion growth and has a favorable safety profile.6 Sustained efficacy of ACP, as shown in the restriction of lesion growth over time, was observed over a follow up of two years in GATHER1 and GATHER2 studies.7
This submission will have no impact on the financial forecasts of the current fiscal year ending March 31, 2025.
About avacincaptad pegol
Avacincaptad pegol (ACP) is an investigational drug for treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) that has been submitted for Conditional Approval in
About Geographic Atrophy
Age-related macular degeneration (AMD) is the major cause of moderate and severe loss of central vision in aging adults, affecting both eyes in the majority of patients.1,8,9 The macula is a small area in the central portion of the retina responsible for central vision. As AMD progresses, the loss of retinal cells and the underlying blood vessels in the macula results in marked thinning and/or atrophy of retinal tissue.10 Geographic atrophy (GA), associated with AMD, leads to further irreversible loss of vision in these patients.11
About the GATHER2 Clinical Trial
GATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD.6 ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly.6 In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP dosed monthly (EM, n=96) or every other month (EOM, n=93); patients who received sham in year 1 continued to receive sham in year 2 (n=203).7 IZERVAY is continuing to be evaluated in an open-label extension study.
About Astellas
Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. Learn more at www.astellas.com.
Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.
References:
1 Keenan TDL, Cukras CA, Chew EY. Age-Related Macular Degeneration: Epidemiology and Clinical Aspects. Adv Exp Med Biol. 2021;1256:1-31.
2 Apellis. Apellis Receives Approval of SYFOVRE® (pegcetacoplan) in
3 Colijn JM, Liefers B, Joachim N, et al. Enlargement of geographic atrophy from first diagnosis to end of life. JAMA Ophthalmol. 2021;139(7):743–750.
4 Lundeen EA, Saydah S, Ehrlich J, Saaddine J. Self-reported vision impairment and psychological distress in
5 World Health Organization. Blindness and vision impairment. Available at: https://www.who.int/news-room/fact-sheets/detail/blindness-and-visual-impairment. Last accessed: February 2025.
6 IZERVAY™ (avancincaptad pegol intravitreal solution) Prescribing Information. February 2024.
7 Khanani AM, Patel SS, Staurenghi G, et al. GATHER2: Two-Year Data. Presented at AAO 2023 127th Annual Meeting.
8 Desai D and Dugel PU. Complement cascade inhibition in geographic atrophy: a review. Eye. 2022;36(2):294–302.
9 Ayoub T and Patel N. Age-related macular degeneration. J R Soc Med. 2009;102(2):56–61.
10 Jaffe GJ, Westby K, Csaky KG, et al. C5 Inhibitor avacincaptad pegol for geographic atrophy due to age-related macular degeneration: a randomized pivotal phase 2/3 trial. Ophthalmology. 2021;128(4):576–586.
11 Patel PJ, Ziemssen F, Ng E, et al. Burden of illness in geographic atrophy: a study of vision-related quality of life and health care resource use. Clin Ophthalmol. 2020;14:15–28.
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SOURCE Astellas Pharma Inc.
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