Iveric Bio announced that the US Food and Drug Administration (FDA) has 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). The NDA has been granted priority review, meaning the FDA will act on the application within 6 months, rather than the 10 months typical under standard review.
By targeting C5, avacincaptad pegol has the potential to decrease activity of the complement system that causes the degeneration of retinal cells and potentially slow the progression of geographic atrophy, the company said in a news release. The FDA had previously granted Breakthrough Therapy status for avacincaptad pegol in November 2022.
“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, chief executive officer of Iveric Bio, said in a news release.
Currently, neither the FDA nor the European Medicines Agency have approved any treatment options for geographic atrophy secondary to AMD, which leads to progressive and irreversible vision loss. Under priority review, the FDA’s Prescription Drug User Fee Act (PDUFA) goal date is August 19, 2023. Iveric Bio noted that the FDA has not identified any potential review issues and that the agency is not currently planning to hold an Advisory Committee meeting for ACP.
Iveric’s NDA was based on the 12-month prespecified primary efficacy and safety results from the phase 3 GATHER1 and GATHER2 clinical trials. These randomized, double-masked, sham-controlled, multicenter 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. 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 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 choroidal neovascularization (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.
“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,” said Pravin U. Dugel, president of Iveric Bio. “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.”