Apellis Announces FDA Acceptance and Priority Review of the Supplemental New Drug Application for EMPAVELI® (pegcetacoplan) for C3G and Primary IC-MPGN
Apellis Pharmaceuticals (APLS) announced FDA acceptance and Priority Review designation for EMPAVELI® (pegcetacoplan) supplemental New Drug Application (sNDA) for treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare kidney diseases. The PDUFA target date is set for July 28, 2025.
The submission is supported by positive Phase 3 VALIANT study results, which showed:
- 68% reduction in proteinuria compared to placebo (p<0.0001)
- Stabilization of kidney function measured by eGFR (p=0.03)
- 71% of treated patients achieved complete clearance of C3c staining
Results were consistent across C3G and IC-MPGN patients, including adolescents, adults, and those with native and post-transplant kidney disease. The treatment demonstrated favorable safety and tolerability.
Apellis Pharmaceuticals (APLS) ha annunciato l'accettazione da parte della FDA e la designazione di Revisione Prioritaria per la domanda di nuovo farmaco supplementare (sNDA) di EMPAVELI® (pegcetacoplan) per il trattamento della glomerulopatia da C3 (C3G) e della glomerulonefrite membranoproliferativa da complessi immunitari primaria (IC-MPGN), malattie renali rare. La data obiettivo PDUFA è fissata per il 28 luglio 2025.
La presentazione è supportata dai risultati positivi dello studio di Fase 3 VALIANT, che hanno mostrato:
- riduzione del 68% della proteinuria rispetto al placebo (p<0.0001)
- Stabilizzazione della funzione renale misurata dall'eGFR (p=0.03)
- 71% dei pazienti trattati ha raggiunto una completa eliminazione della colorazione C3c
I risultati sono stati coerenti tra i pazienti con C3G e IC-MPGN, inclusi adolescenti, adulti e coloro che presentano malattia renale nativa e post-trapianto. Il trattamento ha dimostrato una sicurezza e tollerabilità favorevoli.
Apellis Pharmaceuticals (APLS) anunció la aceptación por parte de la FDA y la designación de Revisión Prioritaria para la Solicitud de Nuevo Medicamento Suplementaria (sNDA) de EMPAVELI® (pegcetacoplan) para el tratamiento de la glomerulopatía por C3 (C3G) y la glomerulonefritis membranoproliferativa por complejos inmunitarios primaria (IC-MPGN), enfermedades renales raras. La fecha objetivo PDUFA está establecida para el 28 de julio de 2025.
La presentación está respaldada por los resultados positivos del estudio de Fase 3 VALIANT, que mostraron:
- reducción del 68% en la proteinuria en comparación con el placebo (p<0.0001)
- Estabilización de la función renal medida por eGFR (p=0.03)
- 71% de los pacientes tratados lograron una eliminación completa de la tinción C3c
Los resultados fueron consistentes entre los pacientes con C3G e IC-MPGN, incluidos adolescentes, adultos y aquellos con enfermedad renal nativa y post-trasplante. El tratamiento demostró una seguridad y tolerabilidad favorables.
Apellis Pharmaceuticals (APLS)는 FDA의 수용과 EMPAVELI® (pegcetacoplan)의 보충 신약 신청(sNDA)에 대한 우선 검토 지정 발표를 했습니다. 이는 C3 사구체병(C3G) 및 원발 면역 복합체 막 증식성 사구체신염(IC-MPGN)이라는 희귀 신장 질환 치료를 위한 것입니다. PDUFA 목표 날짜는 2025년 7월 28일로 설정되어 있습니다.
이 제출은 긍정적인 3상 VALIANT 연구 결과에 의해 뒷받침됩니다. 결과는 다음과 같습니다:
- 68%의 단백뇨 감소, 위약 대비 (p<0.0001)
- eGFR로 측정한 신장 기능의 안정화 (p=0.03)
- 71%의 치료 환자가 C3c 염색의 완전 제거를 달성함
결과는 C3G 및 IC-MPGN 환자들, 청소년, 성인 및 원래 신장 질환과 이식 후 신장 질환 환자들 사이에서 일관되었습니다. 이 치료는 유리한 안전성과 내약성을 보여주었습니다.
Apellis Pharmaceuticals (APLS) a annoncé l'acceptation par la FDA et la désignation de Révision Prioritaire pour la Demande de Médicament Nouveau Supplémentaire (sNDA) d'EMPAVELI® (pegcetacoplan) pour le traitement de la glomérulopathie C3 (C3G) et de la glomérulonéphrite membranoproliférative à complexes immunitaires primaire (IC-MPGN), des maladies rénales rares. La date cible PDUFA est fixée au 28 juillet 2025.
La soumission est soutenue par des résultats positifs de l'étude de Phase 3 VALIANT, qui ont montré :
- réduction de 68% de la protéinurie par rapport au placebo (p<0.0001)
- Stabilisation de la fonction rénale mesurée par eGFR (p=0.03)
- 71% des patients traités ont atteint une élimination complète de la coloration C3c
Les résultats étaient cohérents chez les patients atteints de C3G et d'IC-MPGN, y compris les adolescents, les adultes et ceux atteints de maladies rénales natives et post-transplantation. Le traitement a montré une sécurité et une tolérance favorables.
Apellis Pharmaceuticals (APLS) hat die Annahme durch die FDA und die Einstufung als Prioritätsprüfung für den ergänzenden Antrag auf Zulassung eines neuen Arzneimittels (sNDA) für EMPAVELI® (pegcetacoplan) zur Behandlung von C3-Glomerulopathie (C3G) und primärer immuner Komplex-membranproliferativer Glomerulonephritis (IC-MPGN), seltene Nierenerkrankungen, bekannt gegeben. Das PDUFA-Zieldatum ist auf den 28. Juli 2025 festgelegt.
Die Einreichung wird durch positive Ergebnisse der Phase-3-VALIANT-Studie unterstützt, die Folgendes zeigten:
- 68% Reduktion der Proteinurie im Vergleich zu Placebo (p<0.0001)
- Stabilisierung der Nierenfunktion gemessen durch eGFR (p=0.03)
- 71% der behandelten Patienten erreichten eine vollständige Beseitigung der C3c-Färbung
Die Ergebnisse waren konsistent bei Patienten mit C3G und IC-MPGN, einschließlich Jugendlichen, Erwachsenen und Personen mit nativer und posttransplantierter Nierenerkrankung. Die Behandlung zeigte eine günstige Sicherheit und Verträglichkeit.
- FDA granted Priority Review designation, potentially expediting approval process
- Strong Phase 3 trial results with 68% proteinuria reduction
- 71% of patients achieved complete C3c staining clearance
- Demonstrated efficacy across all patient subgroups
- Favorable safety and tolerability profile
- Full FDA approval still pending until July 2025
Insights
The FDA's Priority Review designation for Apellis' EMPAVELI sNDA represents a significant regulatory milestone that substantially accelerates the review timeline from 10 months to 6 months. With a PDUFA date of July 28, 2025, the company now has a clear regulatory pathway and timeline for potential approval.
Priority Review is only granted when the FDA determines a therapy could provide significant improvements in treatment efficacy or safety for serious conditions. This designation itself validates the clinical significance of EMPAVELI's potential in these rare kidney diseases and suggests the agency recognizes the substantial unmet need.
The comprehensive Phase 3 VALIANT data package supporting this application is particularly robust, demonstrating statistically significant benefits across multiple clinically relevant endpoints. Most notably, the application covers broad patient populations regardless of disease type, age, or transplant status - eliminating potential limitations in the label that could restrict market access.
The regulatory strategy of pursuing approval for both C3G and IC-MPGN simultaneously is shrewd, as these ultra-rare diseases individually represent small patient populations, but collectively create a more commercially viable opportunity. If approved, Apellis would secure market exclusivity protections while addressing conditions with virtually no approved therapies.
The clinical data from the VALIANT trial represents genuinely breakthrough efficacy for C3G and IC-MPGN patients who currently have extremely treatment options. The
Most impressive is the constellation of benefits across all three critical disease markers. Proteinuria reduction alone would be noteworthy, but the concurrent stabilization of kidney function (eGFR) and the remarkable
The mechanism of action targeting C3 makes perfect biological sense for these complement-mediated nephropathies. By inhibiting the central component of the complement cascade, pegcetacoplan addresses the fundamental pathophysiology driving disease progression.
For nephrologists treating these patients, the potential approval would represent the first therapy specifically validated for these conditions. Currently, patients often progress to end-stage kidney disease despite off-label immunosuppression. The kidney-sparing potential demonstrated here could fundamentally alter the natural history of these diseases, potentially reducing transplantation needs and improving long-term outcomes across diverse patient populations.
- PDUFA target action date is July 28, 2025
WALTHAM, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases. The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025.
“EMPAVELI demonstrated clinically meaningful benefits across all three key markers of disease – unprecedented proteinuria reductions, stabilization of kidney function, and substantial clearance of C3c staining,” said Bradley P. Dixon, M.D., FASN, professor of pediatrics and medicine, University of Colorado School of Medicine, and co-investigator of the VALIANT study. “Efficacy is critically important when treating C3G and IC-MPGN given the high risk of progression to kidney failure. As a disease-modifying therapy, EMPAVELI has the potential to make a life-changing difference for patients, if approved.”
“This milestone represents a significant step toward our goal of bringing EMPAVELI to people living with C3G and primary IC-MPGN, regardless of their disease type, age, or transplant status,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder at Apellis. “We look forward to working with the FDA to make this treatment available to patients in need as quickly as possible.”
sNDA submission supported by positive Phase 3 VALIANT results at Week 26
The positive results were consistent across patients with C3G and IC-MPGN, adolescents and adults, and native and post-transplant kidney disease.
- Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant
68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo. - Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function (nominal p=0.03) as measured by eGFR.
- Reduction of C3c staining: A substantial proportion of EMPAVELI-treated patients achieved a reduction in C3c staining intensity (nominal p<0.0001).
71% of EMPAVELI-treated patients showed complete clearance of C3c staining compared to placebo.
EMPAVELI showed favorable safety and tolerability, consistent with its established profile.
Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition.
About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately
About the VALIANT Study
The VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.
About Pegcetacoplan in Rare Diseases
Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across hematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally.
About the Apellis and Sobi Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
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 many serious diseases. For more information, please visit http://apellis.com or follow us on X (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. These statements include, but are not limited to, statements regarding plans to submit applications for regulatory approval for the treatment of patients with C3G and IC-MPGN. 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 systemic pegcetacoplan will receive approval for those indications from the FDA or equivalent foreign regulatory agencies when expected or at all; and any other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 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:
Lissa Pavluk
media@apellis.com
617-977-6764
Investors:
Meredith Kaya
meredith.kaya@apellis.com
617-599-8178
References
1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.
2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474.
3. Data on file using literature consensus.
FAQ
What were the key results of EMPAVELI's Phase 3 VALIANT trial for C3G and IC-MPGN?
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