Iveric Bio submitted to the FDA the third and final part of its new drug application (NDA) for rolling review of Zimura (avacincaptad pegol) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Tell me about avacincaptad pegol.
Avacincaptad pegol (ACP) is a novel investigational complement C5 inhibitor — overactivity of C5 protein and the complement system are thought to have a significant role in scarring and vision loss associated with GA secondary to AMD. By targeting C5 protein, ACP may decrease activity in the complement system that leads to retinal cell degeneration and, as a result, slow GA progression. (via)
How did it perform in clinical trials?
The GATHER1 (NCT02686658) and GATHER2 (NCT04435366) phase 3 clinical trials evaluated the safety and efficacy of 2 mg monthly intravitreal injections of ACP compared to sham. Twelve-month data from the trials found ACP to be the only investigational product for treating GA that achieved its pre-specified 12-month primary endpoint, with efficacy rates observed up to 35%.
Any adverse events?
The most frequently reported reactions (≥ 5% and greater than sham) were conjunctival hemorrhage (13%); increased intraocular pressure (9%); and choroidal neovascularization (7%).
No serious reactions were reported after 12 months in GATHER1 and 18 months in GATHER2.
What makes it unique?
ACP is the first and currently only investigational therapy that has been granted the FDA’s Breakthrough Therapy designation for GA secondary to AMD.
What’s next?
Stay tuned! The FDA now has 60 days to consider the application for review. If accepted, a Prescription Drug User Fee Act (PDUFA) may come next summer.