GA patients experienced 36% reduction in lesion growth in the 18-24 month period of Syfovre's FDA trials. Clinical images are bilateral GA in a 90-year-old not part of the FDA trials. Photos: Apellis (product image); Anna Bedwell, OD (clinical photos).

Retina specialists will soon be able to treat patients with geographic atrophy (GA) in hopes of mitigating the loss of retinal cells and subsequent visual impairment that often develops in those with dry AMD. On Friday the FDA approved the use of pegcetacoplan, a C3 inhibitor, from Apellis Pharmaceuticals for this indication. It will be marketed under the brand name Syfovre and will be priced at $2,190 per vial, the company announced on Friday.

In an investor call on Friday afternoon,  Chief Medical Officer Caroline Baumal estimated that patients will need six to eight vials of Syfovre per year. A program called ApellisAssist will provide insurance support, financial assistance and disease education for eligible patients, the company said in the release.

Syfovre is approved for GA patients with or without subfoveal involvement and should be administered every 25 to 60 days, a flexible dosing schedule that should help patients and doctors alike maintain therapy over the long haul, the company noted.

Patients need to commit to an extended course of therapy to experience the drug’s benefits. “This is not a drug that flips the switch on day one,” Apellis CEO Cedric Francois, MD, PhD, told Fierce Pharma in a recent interview. “It’s a drug where you make an investment over many years, and where the longer you stick to the therapy, the better this drug is going to work for you.” 

In its FDA clinical trials (named OAKS and DERBY), the drug “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,” according to the press release. OAKS and DERBY subjects are currently participating in three-year extension trials to gauge the durability of treatment effect out to five years.

More than one million Americans have developed GA, Apellis says. The condition arises from the combined effects of genetic influencers, environmental stressors and the aging process. Central to the pathogenesis is the complement cascade, an immune response in which a protein compound called membrane attack complex is created, which then adheres to cells and causes lysis and cell death. Pegcetacoplan blocks the cleavage of complement protein C3 into C3a and C3b; the latter is an upstream precursor of membrane attack complex.

Apellis says that Syfovre availability will begin in March. For more information, visit https://syfovreecp.com.