Iveric Bio announced a PDUFA goal date of Aug. 19, 2023. The FDA has not identified any potential review issues and is not currently planning to hold an Advisory Committee meeting for avacincaptad pegol.
Iveric Bio announced the FDA has completed its filing review and accepted the company’s New Drug Application (NDA) for avacincaptad pegol (ACP), a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to Age-Related Macular Degeneration (AMD).
According to a news release from the company, the NDA has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) goal date of August 19, 2023. The company also announced the FDA has not identified any potential review issues and the FDA is not currently planning to hold an Advisory Committee meeting for ACP.
“The FDA’s acceptance of our NDA and Priority Review for avacincaptad pegol bring us another significant step closer to delivering a much-needed treatment to AMD patients living with GA,” Glenn P. Sblendorio, CEO of Iveric Bio, said in the news release. “We look forward to continuing our collaboration with the FDA throughout the review process.”
The company pointed out in its news release that he NDA submission is based on the 12-month pre-specified primary efficacy and safety results from the GATHER1 and GATHER2 clinical trials. ACP is the only investigational product for the treatment of GA to achieve the pre-specified 12-month primary endpoint in two phase 3 pivotal trials with observed efficacy rates of up to 35%. In addition, ACP is the first and only investigational therapy to receive Breakthrough Therapy designation for GA secondary to AMD.
According to the company, 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.
ACP met its primary endpoint in the completed GATHER1 clinical trial and the ongoing GATHER2 clinical trial both of which are randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trials. These clinical trials evaluated the safety and efficacy 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.
“We believe our Special Protocol Assessment for GATHER2, rolling review, Breakthrough Therapy designation and now Priority Review underscore the strength of our GATHER1 and GATHER2 results,” Pravin U. Dugel, president of Iveric Bio said in a statement. “We continue to accelerate our commercial launch plans and prepare for a potential approval of ACP for the treatment of GA throughout the AMD disease continuum. This is important because AMD leads to irreversible, and in many cases catastrophic, vision loss.”
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 company noted that 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 recommended 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.