IVERIC bio, Inc., (NASDAQ: ISEE), based in Parsippany New Jersey, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for avacincaptad pegol (ACP, known as “Zimura”), a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). According to the company, ACP is the first and only investigational therapy to receive Breakthrough Therapy designation status for this indication, which was granted based on the 12-month pre-specified primary endpoint data in the GATHER1 and GATHER2 pivotal clinical trials.
FDA Breakthrough Therapy designation is aimed to accelerate the development and regulatory review of potential new medicines intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to show preliminary clinical evidence that the drug may demonstrate substantial improvement in effectiveness or safety over available therapies on a clinically significant endpoint. The decision by the FDA was based on the 12-month primary efficacy endpoint data from the GATHER1 and GATHER2 pivotal studies, which evaluated the safety and efficacy of ACP in patients with GA located inside and/or outside of the clinical fovea. Following a special protocol assessment (SPA) agreement for GATHER2, the FDA required the mean rate of growth in GA area from baseline to month 12. The results showed a significant treatment difference of 35% (P=0.0050; GATHER1) and 18% (P=0.0039, GATHER2) compared to sham using observed (non-transformed) data; and 28% (P=0.0063; GATHER1) and 14% (P=0.0064; GATHER2) using square root transformation. In both GATHER1 and GATHER2 there were no events of serious intraocular inflammation, vasculitis, or endophthalmitis.
According to the company, Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio., stated that “we believe this Breakthrough designation reflects the fact that both GATHER1 and GATHER2 met their primary endpoint with a safety profile that meets the stringent criteria required. We are now focused on the execution of our full NDA submission and launch preparation, with the possibility of being first to market. We look forward to working collaboratively with the FDA to expedite the review timeline for avacincaptad pegol and to potentially bringing a new therapy to AMD patients impacted by GA.” In additionally, Pravin U. Dugel, MD, President of Iveric Bio., commented that, “in both GATHER1 and GATHER2, avacincaptad pegol consistently showed a treatment effect with the first measurement at month 6 that was persistent and continued to increase over time, with observed efficacy rates of up to 35%. We believe ACP has the potential to safely and effectively preserve central vision by saving photoreceptor cells for patients living with this life-changing disease that leads to irreversible blindness.”