Apellis Announces Submission of Pegcetacoplan Marketing Applications to FDA and EMA for Patients with PNH
Apellis Pharmaceuticals (Nasdaq: APLS) has submitted a New Drug Application to the FDA and a Marketing Authorization Application to the EMA for pegcetacoplan, targeting paroxysmal nocturnal hemoglobinuria (PNH). The submissions rely on successful Phase 3 PEGASUS study results, showing pegcetacoplan's superiority to eculizumab in improving hemoglobin levels and other key metrics. Additionally, Australian regulators granted orphan drug designation for pegcetacoplan. FDA and EMA decisions on the submissions are anticipated in Q4 2020.
- Submission of NDA and MAA for pegcetacoplan to FDA and EMA.
- Phase 3 PEGASUS study demonstrated pegcetacoplan's superiority to eculizumab in hemoglobin improvement.
- Australian TGA granted orphan drug designation for pegcetacoplan in PNH.
- None.
WALTHAM, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States and European Union, respectively.
“Pegcetacoplan has demonstrated its potential to elevate the standard of care in PNH and the submissions of the U.S. and EU marketing applications represent an important step in our efforts to bring the first targeted C3 therapy to people with PNH as quickly as possible,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “Building on our progress with the FDA and EMA, we are also proud that the TGA has granted pegcetacoplan orphan drug designation for the treatment of patients with PNH in Australia.”
The NDA and MAA submissions are based on results from the Phase 3 PEGASUS study, which met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks, as well as higher normalization rates across key markers of hemolysis and clinically meaningful improvement in FACIT-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study. The FDA and EMA decisions on acceptance of the NDA and MAA submissions are expected in Q4 2020.
In another regulatory milestone, the Australian Therapeutic Goods Administration (TGA) granted orphan drug designation to pegcetacoplan in PNH. This designation in Australia is granted to therapies for serious rare diseases that have the potential to provide a significant benefit in comparison to approved treatments. Apellis plans to submit a marketing application in Australia in the fourth quarter of 2020.
About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea). Retrospective studies show that, even on eculizumab, approximately
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 targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.
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 relating to the implications of preliminary clinical data. 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 company’s clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company’s clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020 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:
Mark Dole
media@apellis.com
617.420.4839
Investor Contact:
Sam Martin / Maghan Meyers
Argot Partners
sam@argotpartners.com / maghan@argotpartners.com
212.600.1902
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1. Risitano AM, Notaro R, Marando L, et al. (2009) Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009 Apr 23;113(17):4094-100.
2. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.
FAQ
What recent regulatory submissions has Apellis Pharmaceuticals made for pegcetacoplan?
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