FDA Approves SYFOVRE™ (pegcetacoplan injection) as the First and Only Treatment for Geographic Atrophy (GA), a Leading Cause of Blindness
- SYFOVRE slowed GA progression with increasing effects over time
- Approved for all patients with GA, with dosing flexibility every 25 to 60 days
- Well-demonstrated safety profile following ~12,000 injections over 24 months
- Conference call today at 4:30 p.m. ET
A Media Snippet accompanying this announcement is available by clicking on the image or link below:
WALTHAM, Mass., Feb. 17, 2023 (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 approved SYFOVRE™ (pegcetacoplan injection) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). SYFOVRE is the first and only FDA-approved treatment for GA, a leading cause of blindness that impacts more than one million people in the U.S. and five million people worldwide.1,2
“The approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, M.D., Ph.D., lead investigator for the OAKS study, director of ophthalmology clinical research, associate professor of ophthalmology, Duke University Medical Center. “Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined. With SYFOVRE, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”
“Today marks an extraordinary milestone for patients, the retina community, and Apellis. With its increasing effects over time and flexible dosing, we believe that SYFOVRE will make a meaningful difference in the lives of people with GA,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. “GA is a complex disease that the field has spent decades trying to address, so we are humbled and proud to bring forward the first-ever treatment. Thank you to all who helped make this moment a reality.”
The approval of SYFOVRE is based on positive results from the Phase 3 OAKS and DERBY studies at 24 months across a broad and representative population of patients. SYFOVRE is approved for GA patients with or without subfoveal involvement and provides dosing flexibility for patients and physicians with a dosing regimen of every 25 to 60 days. In the OAKS and DERBY studies, SYFOVRE reduced the rate of GA lesion growth compared to sham and demonstrated increasing treatment effects over time, with the greatest benefit (up to 36% reduction in lesion growth with monthly treatment in DERBY) occurring between months 18-24.
“For the first time ever, we are celebrating the approval of a treatment for GA,” said Jeff Todd, president and chief executive officer, Prevent Blindness. “This is a historic and hopeful day for all GA patients and their care partners, who have been waiting for a treatment for this relentless form of vision loss.”
GA is an advanced form of AMD. 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
The safety profile of SYFOVRE is well-demonstrated following ~12,000 injections. The most common adverse reactions (≥ 5%) reported in patients receiving SYFOVRE were ocular discomfort, neovascular AMD, vitreous floaters, and conjunctival hemorrhage.
Apellis is committed to helping patients with treatment access and support. ApellisAssist® is a program designed to help SYFOVRE patients along their treatment journey by providing a system inclusive of insurance support, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information.
SYFOVRE is expected to be available by the beginning of March through specialty distributors and specialty pharmacies nationwide. A marketing authorization application for SYFOVRE is under review by the European Medicines Agency with a decision expected in early 2024. In addition, a marketing application has been submitted to Health Canada.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss the FDA’s approval of SYFOVRE today, Friday, February 17, 2023 at 4:30 p.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website. A replay of the webcast will be available for 30 days following the event.
About the Phase 3 OAKS and DERBY Studies
OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of SYFOVRE™ (pegcetacoplan injection) with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated the efficacy of monthly and every-other-month SYFOVRE in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 24 months.
Both monthly and every-other-month (EOM) SYFOVRE reduced the rate of GA lesion growth through 24 months compared to sham:
- OAKS: 22% monthly; 18% EOM
- DERBY: 18% monthly; 17% EOM
About SYFOVRE™ (pegcetacoplan injection)
SYFOVRE™ (pegcetacoplan injection) is the first and only approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration.
U.S. Important Safety Information for SYFOVRE™ (pegcetacoplan injection)
- SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation
WARNINGS AND PRECAUTIONS
- Endophthalmitis and Retinal Detachments
- Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately.
- Neovascular AMD
- In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration.
- Intraocular Inflammation
- In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE.
- Increased Intraocular Pressure
- Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed.
- Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage.
Please see accompanying full Prescribing Information for more information.
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 and only therapy for geographic atrophy, a leading cause of blindness around the world. With nearly a dozen clinical and pre-clinical programs underway, 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 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 timing of the commercial availability of SYFOVRE. 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 SYFOVRE will be commercially available when expected; whether clinical trials of SYFOVRE indicate an apparent positive effect that is greater than the actual positive effect, whether SYFOVRE will receive approval from 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.
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.
2 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.
3 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
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