Apellis Pharmaceuticals has received FDA approval for Syfovre (pegcetacoplan), indicated for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
What is Syfovre?
Syfovre contains pegcetacoplan, which has been investigated as a targeted C3 inhibition therapy. It works by regulating excessive activation of the complement cascade, which could lead to the onset and progression of diseases.
How does it work?
The formulation is designed as a 15 mg/ml dose to be administered by intravitreal injection once every 25 to 60 days into each affected eye to reduce the progression of GA.
Give me some background.
The FDA granted intravitreal pegcetacoplan (Syfovre) Fast Track designation in 2018 (for GA secondary to AMD). Of note, Apellis already has another medication containing the same molecule called Empaveli, which is approved for paroxysmal nocturnal hemoglobinuria (PNH).
What did clinical trials show?
The DERBY and OAKS studies compared Syfovre’s efficacy and safety with the use of sham injections. Investigators tracked the efficacy of monthly and bimonthly Syfovre in patients with GA assessed by change in the total area of GA lesions from baseline measured by fundus autofluorescence at 12 months. Patients continued to receive masked treatment for 24 months. (via)
Both monthly and bimonthly injections showed a clinically meaningful reduction—36% in DERBY and 29% in OAKS—in GA lesion growth when compared to the sham injections from months 18 to 24.
How about adverse reactions?
The most common adverse reactions (AEs) reported in ≥ 5% of patients included ocular discomfort, neovascular AMD, vitreous floaters, and conjunctival hemorrhage.
When can my patients start receiving treatment?
Syfovre is expected to be made available in specialty pharmacies and distributors throughout the US in March.
Significance?
This approval marks a historic first for treating patients with GA secondary to AMD. Considering the known permanence of visual loss due to GA, Syfovre has the potential to slow or stop this progressive disease from getting worse—giving patients new hope with the new treatment opportunity.