Apellis Pharmaceuticals announced that the FDA has granted fast track designation to the company’s APL-2, a novel inhibitor of complement factor C3 as a next generation monotherapy, for the treatment of patients with geographic atrophy (GA).
The FDA’s fast track program facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need, allowing important new drugs to reach the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may have potential benefits over existing therapies. Drugs that receive a Fast Track designation are eligible for more frequent meetings and written communication with the FDA to discuss development plans and clinical trial design.
“The Fast Track designation represents an important recognition by the FDA of APL-2’s potential to treat geographic atrophy, a blinding disease for which there is no treatment,” Cedric Francois, MD, PhD, founder and chief executive officer of Apellis, said in a company news release. “The severe vision loss associated with GA makes it difficult for patients to recognize faces, read, drive a car and go about their daily lives, limiting quality of life. We believe that by slowing down the rate of degeneration through broad C3 inhibition, we may be able to improve outcomes for these patients.”
Apellis plans to initiate its phase 3 program for patients with GA later this year which will consist of two identical, prospective, multicenter, randomized, double-masked, sham-injection controlled studies to assess the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with GA.
About the FILLY trial
The FILLY trial is a 246-patient phase 2 multicenter, randomized, single-masked, sham-controlled clinical trial of APL-2 in patients with GA conducted at over 40 clinical sites, located in the United States, Australia, and New Zealand. APL-2 was administered as an intravitreal injection in the study eye monthly or every other month for 12 months, followed by 6 months of monitoring without active treatment until month 18. Eyes were evaluated for GA by fundus autofluorescence photographs (FAF). The rate of GA area growth was measured from baseline to month 18. The primary efficacy endpoint was the change in GA lesion size from baseline to month 12, compared to sham.