Iveric Bio announced that the FDA has granted Breakthrough Therapy designation for 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).
To date, ACP is the first and only investigational therapy to receive Breakthrough Therapy designation status for this indication. The 12-month prespecified primary endpoint data from the pivotal GATHER1 and GATHER2 clinical trials served as the basis for the designation of ACP.
Breakthrough Therapy designation is designed to accelerate the development and regulatory review of potential new medicines that are 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 and in general, should show a clear advantage.
“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,” Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio, said in a company news release. “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.”
Iveric Bio recently announced the rolling review submission of the first part of its new drug application (NDA), which comprised the complete clinical data package from the pivotal GATHER1 and GATHER2 trials. By the end of this year, the company expects to complete the last component of the NDA application.
“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%,” said Pravin U. Dugel, MD, President of Iveric Bio. “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.”
1. U.S. Food and Drug Administration. “Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics, 2014”. Available at https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics. Last accessed: November 16, 2022.