The FDA recently approved a therapy previously approved in 2021….. but with a very different indication and a new NDC. The approval was granted for Syfovre (pegcetacoplan injection) from Apellis Pharmaceuticals, Inc for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). It is the first and only therapy approved to treat geographic atrophy. It is approved for intravitreal administration only by a qualified physician.
This is the second approval for the drug. The FDA first approved pegcetacoplan in paroxysmal nocturnal hemoglobinuria, a rare blood disorder where the immune system attacks red blood cells and platelets. It is marketed as Empaveli in that indication.
Although the approval did not include a black box warning the therapy can trigger ocular and periocular infections, active intraocular inflammation, endophthalmitis and retinal detachments among other adverse events.
The recommended dose for Syfovre is 15 mg (0.1 mL of 150 mg/mL solution) administered by intravitreal injection to each affected eye once every 25 to 60 days.
The injection should be on the market by the beginning of March. The company announced launch price at $2,190 per vial before discounts. That translates into between $13,200 and $26,400 annually based on 30 vs. 60-day dosing.
Apellis did not announce distribution plans for Syfovre. Given its cost it is likely that it will be distributed by a specialty pharmacy distributor.
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US FDA approves Apellis’ geographic atrophy therapy Syfovre
Syfovre is indicated for geographic atrophy patients with or without subfoveal involvement.
The US Food and Drug Administration (FDA) has granted approval for Apellis Pharmaceuticals’ Syfovre (pegcetacoplan injection) to treat geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD).
Syfovre has been approved for use in people with GA with or without subfoveal involvement and offers to dose flexibility with a regimen of every 25 to 60 days for patients and physicians. It provides comprehensive control of the complement cascade, which is part of the immune system of the body, by targeting C3.
Apellis Pharmaceuticals co-founder and CEO Cedric Francois said: “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. 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.”
The regulatory approval is based on positive data from the Phase III OAKS and DERBY trials conducted across a broad and representative population of GA patients. Syfovre reduced the GA lesion growth rate compared to sham injections in the OAKS and DERBY studies.
It also showed increasing effects of treatment over time, with up to 36% lesion growth reduction occurring between months 18 and 24. Syfovre’s safety profile is well-demonstrated after approximately 12,000 injections over 24 months.
