Apellis Pharmaceuticals announced that the FDA has accepted and given Priority Review status to its treatment for geographic atrophy. (via)
What is the treatment?
The investigational drug is pegcetacoplan, which works by inhibiting C3, a central protein in the complement cascade. The complement system is part of the immune system, which defends the body against infection. An overactivation of the complement system can cause destruction of healthy cells and tissue and can contribute to the pathogenesis of AMD.
What did the clinical trials show?
The NDA submission is based on results from the phase 3 DERBY and OAKS studies at 12 and 18 months and the phase 2 FILLY study at 12 months.
In the studies, treatment with both monthly and every-other-month pegcetacoplan resulted in a clinically meaningful reduction of GA lesion growth across a broad, heterogeneous population of more than 1,500 patients. Pegcetacoplan demonstrated a favorable safety profile in all three studies.
When will we hear more?
Later this year. The PDUFA target action date (the date by which the FDA must complete the review process) is set for November 26th.
Want to better understand the complement pathway?
Here’s an excellent explanation in this video.