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PDUFA target action date is November 26, 2022
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FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application
WALTHAM, Mass., July 19, 2022 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation for the intravitreal pegcetacoplan New Drug Application (NDA). Pegcetacoplan is an investigational, targeted C3 therapy for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The Prescription Drug User Fee Act (PDUFA) target action date is November 26, 2022. The FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.
“This is a very exciting time for the retina community as we are closer than ever to having the first treatment for GA, an irreversible and devastating disease that causes a tremendous amount of vision loss,” said Charles Wykoff, M.D., Ph.D., OAKS study investigator and director of research, Retina Consultants of Texas. “Both monthly and every-other-month pegcetacoplan meaningfully slowed GA disease progression across three large studies, and the potential approval of this medicine would be a breakthrough for patients.”
The NDA submission is based on results from the Phase 3 DERBY and OAKS studies at 12 and 18 months and the Phase 2 FILLY study at 12 months. In the studies, treatment with both monthly and every-other-month pegcetacoplan resulted in a clinically meaningful reduction of GA lesion growth across a broad, heterogeneous population of more than 1,500 patients. Pegcetacoplan demonstrated a favorable safety profile in all three studies.
“This important milestone marks our second NDA acceptance in less than two years, a testament to the potential of targeting C3 to treat serious diseases with significant unmet need,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “We are committed to working closely with the FDA to bring this potential, first-ever treatment to patients living with GA as quickly as possible.”
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. Pegcetacoplan was previously granted Fast Track designation by the FDA for the treatment of GA secondary to AMD.
Apellis plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.
About DERBY and OAKS
DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 12 months. Patients in DERBY and OAKS will continue to receive masked treatment for 24 months. Secondary functional endpoints will be evaluated after all patients have received treatment for 24 months.
About FILLY
The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled study evaluating the efficacy and safety of intravitreal pegcetacoplan in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the study was to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in square root GA lesion size from baseline as measured by fundus autofluorescence at 12 months followed by six months of monitoring after the end of treatment.
About Geographic Atrophy (GA)
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness that impacts more than 5 million people worldwide, including one million people in the United States.
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This progressive disease can severely impair visual function, independence, and quality of life as it takes on average 2.5 years for GA lesions to encroach the fovea, which is responsible for central vision.
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GA is caused by destruction of retinal cells through irreversible lesion growth that is driven by excessive complement activation.
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C3 is the only target that can precisely control the complement cascade due to its central location. There are currently no approved treatments for GA.
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. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, and neurological diseases. For more information, please visit
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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 timing of anticipated regulatory submissions or decisions. 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 the results of the FILLY, DERBY and OAKS trials are sufficient to support regulatory submissions; whether a submission for approval of intravitreal pegcetacoplan for GA on the basis of the FILLY, DERBY and OAKS trials will be accepted by foreign regulatory agencies; whether intravitreal pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA when expected or at all; 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 28, 2022 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.
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617.977.6764
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1
Rudnicka 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.
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Wong 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.
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Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
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Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.
