Iveric Bio Submits First Part of NDA for Rolling Review of Avacincaptad Pegol for the Treatment of Geographic Atrophy
Iveric bio announced that it has submitted to the FDA the first part of its new drug application (NDA) for rolling review of avacincaptad pegol (ACP, also known as Zimura) a novel investigational complement C5 inhibitor, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). As previously announced, the company received Fast Track designation from the FDA for ACP. Following receipt of the topline GATHER2 data, the company shared the data with the FDA. Based on the pivotal clinical trials, GATHER1 and GATHER2, which both met their primary endpoint in slowing GA progression with statistical significance at the 12-month time point, the company requested rolling submission of its planned NDA, which the FDA granted. As per the company’s agreement with the FDA, the first part of the NDA, which included the complete ACP clinical data package, was successfully submitted earlier today.
“We are pleased to report this important milestone and look forward to closely collaborating with the FDA throughout the review of our NDA,” Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio, said in a company news release. “As we stated previously, our Special Protocol Assessment (SPA) agreement with the FDA provides a basis for review of our NDA based on 12-month safety and efficacy results from GATHER2, taken together with the results of GATHER1.”
About Avacincaptad Pegol
Avacincaptad pegol (ACP) is an investigational drug that has not yet been evaluated by any regulatory body for safety and efficacy. ACP is not authorized for any indication in any country. ACP is a novel complement C5 protein inhibitor. Overactivity of the complement system and the C5 protein are suspected to play a critical role in the development and growth of scarring and vision loss associated with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). By targeting C5, ACP has the potential to decrease activity of the complement system that causes the degeneration of retinal cells and potentially slow the progression of GA.
About the GATHER Clinical Trials
The company previously reported that ACP met its primary endpoint in its completed randomized, double-masked, sham-controlled, multicenter GATHER1 clinical trial and its ongoing GATHER2 clinical trial, both of which are phase 3 randomized, double-masked, sham-controlled, multicenter clinical trials. These clinical trials measured the efficacy and safety of monthly 2 mg intravitreal administration of ACP in patients with GA secondary to AMD. For the first 12 months in both trials, patients were randomized to receive either ACP 2 mg or sham monthly. There were 286 participants enrolled in GATHER1 and 448 participants enrolled in GATHER2. The primary efficacy endpoints in both pivotal studies were based on GA area measured by fundus autofluorescence at three time points: Baseline, Month 6, and Month 12. The mean rate of growth (slope) in GA area from baseline to month 12 using observed data was 35% in GATHER 1 and 18% in GATHER2. In GATHER1 and GATHER2 combined, the most frequently reported treatment emergent adverse events in the 2 mg dose were related to injection procedure. The most common adverse reactions (≥ 5% and greater than sham) reported in patients who received avacincaptad pegol 2 mg were conjunctival hemorrhage (13%), increased IOP (9%), and CNV (7%). After 18 months of treatment in GATHER1 and 12 months of treatment in GATHER2, there were no events of serious intraocular inflammation, vasculitis, or endophthalmitis.