Iveric Bio announced that the FDA has accepted its new drug application (NDA) for Zimura (avacincaptad pegol), a novel investigational complement C5 inhibitor, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
This submission follows the company’s initial NDA submission to the FDA in November 2022.
Tell me more about Zimura.
Overactivity within the complement system and the C5 protein are thought to play key roles in the development and growth of scarring and vision loss linked with GA secondary to AMD. By focusing on C5, Zimura may decrease the complement system’s activity that can lead to retinal cell degeneration— thus slowing GA progression.
How did it perform in clinical trials?
Zimura met its primary endpoint in the phase 3 GATHER1 (NCT02686658) and GATHER2 (NCT04435366) clinical trials. Both trials measured the safety and efficacy of 2 mg of Zimura administered intravitreally on a monthly basis for 12 months in patients with GA secondary to AMD. GATHER1 included 286 participants and GATHER2 included 448 participants.
Primary efficacy endpoints were based on GA area measured by fundus autofluorescence at three timepoints: baseline, 6 months, and 12 months. The mean rate of growth in the GA area from baseline to month 12, based on data, was 35% in GATHER1 and 18% in GATHER2.
See here for additional data.
What else?
The FDA also granted Priority Review for Zimura, along with a Prescription Drug User Fee Act (PDUFA) date planned for August 19, 2023.
Significance?
Zimura is currently the only investigational candidate treatment for GA treatment that has achieved its primary efficacy endpoint, and is also the only such therapy to receive Breakthrough Therapy designation for GA secondary to AMD (in November 2022).