Wait … didn’t Iveric Bio just submit a new drug application (NDA) for Zimura?
Yes it did! The company submitted the first part of its NDA to the FDA for rolling review earlier this month. (via)
What’s the difference between an NDA and breakthrough therapy designation?
A breakthrough therapy designation is intended to expedite the development and review process for drugs designed to treat a serious or life-threatening disease. In comparison, a NDA is when a company formally asks the FDA to consider a specific drug for market approval.
Refresh me on avacincaptad pegol.
Avacincaptad pegol (ACP) is a novel complement C5 inhibitor administered via intravitreal injections into the eye.
Overactivity of the complement system and C5 protein are thought to be key factors leading to the development and growth of scarring/vision loss that’s associated with GA secondary to AMD.
ACP focuses on C5 to—potentially—decrease activity of the complement system that’s behind retinal cell degeneration and—potentially—slow GA progression.
I need a recap on the clinical trials too.
The GATHER1 and ongoing GATHER2 phase 3 clinical trials measured the safety and efficacy of 2 mg of ACP administered intravitreally to patients on a monthly basis for 12 months.
Both trials met their primary endpoints, which were based on GA area measured by fundus autofluorescence at baseline, month 6, and month 12.
The mean growth rate in the GA area from baseline to month 12 was 35% (GATHER1) and 18% (GATHER2).
Any adverse effects?
Both trials reported injection procedure-related reactions (≥ 5% and greater than sham): conjunctival hemorrhage (13%); increased intraocular pressure (9%); and choroidal neovascularization (7%).
What’s next?
Iveric Bio is working on completing the final part of its NDA submission for ACP by the end of 2022.