FDA Approves First Drug for Geographic Atrophy Due to AMD

The FDA has approved Apellis’ Syfovre (pegcetacoplan) to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Geographic atrophy is an advanced form of age-related macular degeneration and a leading cause of blindness that impacts more than 5 million people worldwide, including 1 million people in the United States.

Syfovre is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system. It is designed to be injected into the back of the eye.

Syfovre will be available by the beginning of March through specialty distributors and specialty pharmacies. It will have a list price of $2,190 per vial, and a spokesperson said the price reflects the Syfovre clinical profile and is in line with recently approved anti-VEGF therapies.

Apellis officials anticipate Medicare to be the dominant payer for patients with geographic atrophy because of the age of diagnosis of these patients. The company is helping patients with treatment access and support. ApellisAssist provides insurance support, financial assistance for eligible patients, disease education, and ongoing product support. “Our priority is to ensure patient access so that all appropriate patients with the potential to benefit from SYFOVRE can receive it,” the spokesperson said.

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. In the OAKS and DERBY studies, Syfovre reduced the rate of geographic atrophy lesion growth compared with 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 and 24.

Eleonora Lad, M.D., Ph.D.

“Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined," 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, said in a press release. “ With Syfovre, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”

In clinical trials, Syfovre was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. Additionally, increased intraocular pressure may occur after injection. The most common adverse reactions are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, and conjunctival hemorrhage.

Related: FDA Grants Priority Review to AMD Therapy

The approval comes just after the FDA granted priority review to another therapy forgeographic atrophy. The FDA granted priority review to Iveric Bio’s new drug application (NDA) for avacincaptad pegol (ACP) to treat patients with geographic atrophy secondary to age-related macular degeneration. The Prescription Drug User Fee Act goal date is Aug. 19, 2023.