Apellis Receives Negative CHMP Opinion for Pegcetacoplan for Geographic Atrophy (GA) in the EU Following Re-Examination
Apellis Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration in the EU.
The company expressed disappointment with the decision, which leaves millions of Europeans with GA without a treatment option. Despite support from the European retina community and dissenting votes from some CHMP members, the negative opinion was upheld. Apellis remains committed to expanding access to pegcetacoplan in other regions globally, focusing on unmet patient needs in the U.S. and elsewhere.
GA is an advanced form of age-related macular degeneration and a leading cause of blindness worldwide, affecting over five million people globally. Pegcetacoplan is approved in the United States under the brand name SYFOVRE® for the treatment of GA secondary to age-related macular degeneration.
Apellis Pharmaceuticals ha annunciato che il Comitato per i Farmaci per Uso Umano (CHMP) dell'Agenzia Europea per i Medicinali (EMA) ha confermato il suo parere negativo sulla domanda di autorizzazione alla commercializzazione di pegcetacoplan intravitreale per il trattamento della atrofia geografica (GA) secondaria alla degenerazione maculare legata all'età nell'UE.
La compagnia ha espresso delusione per la decisione, che lascia milioni di europei con GA senza opzioni di trattamento. Nonostante il supporto della comunità retinica europea e i voti in dissenso di alcuni membri del CHMP, il parere negativo è stato mantenuto. Apellis rimane impegnata ad ampliare l'accesso a pegcetacoplan in altre regioni del mondo, concentrandosi sui bisogni insoddisfatti dei pazienti negli Stati Uniti e altrove.
La GA è una forma avanzata di degenerazione maculare legata all'età ed è una delle principali cause di cecità a livello mondiale, colpendo oltre cinque milioni di persone nel mondo. Pegcetacoplan è approvato negli Stati Uniti con il nome commerciale SYFOVRE® per il trattamento della GA secondaria alla degenerazione maculare legata all'età.
Apellis Pharmaceuticals anunció que el Comité de Medicamentos para Uso Humano (CHMP) de la Agencia Europea de Medicamentos (EMA) ha confirmado su opinión negativa sobre la solicitud de autorización de comercialización de pegcetacoplan intravítreo para el tratamiento de atrofia geográfica (GA) secundaria a la degeneración macular relacionada con la edad en la UE.
La empresa expresó su decepción por la decisión, que deja a millones de europeos con GA sin opciones de tratamiento. A pesar del apoyo de la comunidad retiniana europea y los votos en disidencia de algunos miembros del CHMP, se mantuvo la opinión negativa. Apellis sigue comprometida a expandir el acceso a pegcetacoplan en otras regiones del mundo, centrándose en las necesidades insatisfechas de los pacientes en EE.UU. y en otros lugares.
La GA es una forma avanzada de degeneración macular relacionada con la edad y una de las principales causas de ceguera en el mundo, afectando a más de cinco millones de personas a nivel mundial. Pegcetacoplan está aprobado en Estados Unidos bajo la marca SYFOVRE® para el tratamiento de GA secundaria a la degeneración macular relacionada con la edad.
Apellis Pharmaceuticals는 유럽 의약품 청(EMA)의 의약품 사용에 대한 위원회(CHMP)가 황반 위축(GA) 치료를 위한 삶전주사 페그세타콜란의 판매 허가 신청에 대한 부정적인 의견을 확인했다고 발표했습니다.
회사는 이 결정에 실망감을 표명했으며, 이로 인해 수백만 명의 유럽인들이 GA 치료 옵션이 없게 되었습니다. 유럽 망막 커뮤니티의 지원과 일부 CHMP 위원들의 반대 투표에도 불구하고 부정적인 의견이 유지되었습니다. Apellis는 미국 및 기타 지역에서 환자의 unmet needs에 초점을 맞추어 pegcetacoplan의 접근성을 확장하는 데 전념하고 있습니다.
GA는 노인성 황반변성의 진행된 형태이며, 전 세계적으로 주요한 실명의 원인 중 하나로, 500만 명 이상의 사람들이 영향을 받고 있습니다. pegcetacoplan은 미국에서 노인성 황반변성에 따른 GA 치료를 위해 SYFOVRE®라는 상표로 승인되었습니다.
Apellis Pharmaceuticals a annoncé que le Comité des médicaments à usage humain (CHMP) de l'Agence européenne des médicaments (EMA) a confirmé son avis défavorable concernant la demande d'autorisation de mise sur le marché de pegcetacoplan intravitréen pour le traitement de l'atrophie géographique (GA) secondaire à la dégénérescence maculaire liée à l'âge dans l'UE.
L'entreprise a exprimé sa déception face à cette décision, qui laisse des millions d'Européens atteints de GA sans option de traitement. Malgré le soutien de la communauté rétinienne européenne et les votes dissidents de certains membres du CHMP, l'avis défavorable a été maintenu. Apellis reste déterminée à élargir l'accès à pegcetacoplan dans d'autres régions du monde, en se concentrant sur les besoins non satisfaits des patients aux États-Unis et ailleurs.
GA est une forme avancée de dégénérescence maculaire liée à l'âge et l'une des principales causes de cécité dans le monde, touchant plus de cinq millions de personnes à l'échelle mondiale. Pegcetacoplan est approuvé aux États-Unis sous le nom de marque SYFOVRE® pour le traitement de la GA secondaire à la dégénérescence maculaire liée à l'âge.
Apellis Pharmaceuticals gab bekannt, dass der Ausschuss für Humanarzneimittel (CHMP) der Europäischen Arzneimittel-Agentur (EMA) seine negative Stellungnahme zur Zulassung von intravitrealem Pegcetacoplan zur Behandlung der geografischen Atrophie (GA), die sekundär zur altersbedingten Makuladegeneration auftritt, in der EU bestätigt hat.
Das Unternehmen äußerte Enttäuschung über die Entscheidung, die Millionen von Europäern mit GA ohne Behandlungsoptionen lässt. Trotz der Unterstützung der europäischen Retina-Community und abweichender Stimmen einiger CHMP-Mitglieder wurde die negative Stellungnahme aufrechterhalten. Apellis bleibt engagiert, den Zugang zu Pegcetacoplan in anderen Regionen weltweit zu erweitern und sich auf unzureichend gedeckte Patientenbedürfnisse in den USA und anderswo zu konzentrieren.
GA ist eine fortgeschrittene Form der altersbedingten Makuladegeneration und eine der Hauptursachen für Blindheit weltweit, die über fünf Millionen Menschen weltweit betrifft. Pegcetacoplan ist in den Vereinigten Staaten unter dem Markennamen SYFOVRE® zur Behandlung von GA, die sekundär zur altersbedingten Makuladegeneration auftritt, zugelassen.
- Pegcetacoplan (SYFOVRE®) is approved in the United States for treating geographic atrophy
- The company remains committed to expanding access to pegcetacoplan in other global regions
- CHMP confirmed negative opinion on pegcetacoplan for geographic atrophy in the EU
- Apellis will not be able to provide access to pegcetacoplan for GA patients in the EU
- The decision leaves millions of Europeans with GA without a treatment option
This negative CHMP opinion is a significant setback for Apellis, impacting its potential market expansion and revenue growth in Europe. The EU market represents a substantial opportunity, with an estimated 5 million people affected by GA worldwide. This decision could lead to:
- Reduced revenue projections and potential stock price volatility
- Increased pressure on U.S. sales performance
- Potential reassessment of the company's European strategy
Investors should closely monitor Apellis' ability to navigate regulatory challenges and its strategy to maximize SYFOVRE's potential in other markets. The company's focus on unmet patient needs in the U.S. and other regions globally may help mitigate some of the impact, but the European setback remains a significant concern for the company's growth prospects.
From a medical perspective, this decision is disappointing for European GA patients. SYFOVRE (pegcetacoplan) represents a significant advancement in GA treatment, being the first approved therapy for this condition. The Phase 3 data demonstrated clinically meaningful results, potentially slowing disease progression. The lack of approved treatments in Europe means:
- Continued unmet medical need for GA patients
- options for ophthalmologists to manage this progressive condition
- Potential disparity in treatment access between U.S. and EU patients
This decision highlights the challenges in bringing innovative treatments to market, even when supported by clinical data and the medical community. It may also impact future research and development efforts in this therapeutic area within the EU.
WALTHAM, Mass., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.
“We are deeply disappointed by this outcome, which leaves millions of Europeans with GA without a treatment for this irreversible form of blindness,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder, Apellis. “This CHMP opinion was made despite broad support for pegcetacoplan from the European retina community and multiple dissenting votes by CHMP members who supported a path to approval.”
“We have seen the profound impact this medicine has had for GA patients in the U.S. and remain committed to expanding access to this crucial treatment, focusing on serving unmet patient needs in the U.S. and other regions globally,” Dr. Francois continued.
“It is disappointing to know that we will not be able to provide access to this treatment to GA patients in the EU,” said Frank G. Holz, OAKS and DERBY study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany. “As an ophthalmologist and retina specialist, I have seen how patients with GA lose their ability to read, drive, and even see faces. The Phase 3 data for pegcetacoplan are clinically meaningful and showed the potential to make a difference for EU patients.”
About Geographic Atrophy (GA)
Geographic atrophy (GA) is an advanced form of age-related macular degeneration and a leading cause of blindness worldwide, impacting more than one million Americans and five million people worldwide.1,2 It is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. The vision loss caused by GA severely impairs independence and quality of life by making it difficult to participate in daily activities. On average, it takes only 2.5 years for GA lesions to start impacting the fovea, which is responsible for central vision.3
About Pegcetacoplan for Geographic Atrophy (GA)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. SYFOVRE® (pegcetacoplan injection) is approved in the United States for the treatment of GA secondary to age-related macular degeneration.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether pegcetacoplan will receive approval from foreign regulatory agencies for GA when expected or at all, including the impact on the likelihood and timing of such approvals, and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 27, 2024 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Media Contact:
Tracy Vineis
media@apellis.com
617.420.4839
Investor Contact:
Meredith Kaya
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1Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580.
2Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.
3Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
FAQ
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