FDA grants status following primary endpoint data from pivotal trials treating geographic atrophy
Iveric Bio (Iveric) has announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation status for avacincaptad pegol (ACP).
Also known as Zimura, the therapy is a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
It makes ACP is the only investigational therapy to receive breakthrough therapy designation for this indication. The status was granted based on 12-month pre-specified primary endpoint data yielded from the GATHER1 and GATHER2 pivotal clinical trials. There were 286 participants enroled in the first study and 448 in the second.
“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,” commented Glenn Sblendorio, chief executive officer of Iveric Bio. “We are now focused on the execution of our full NDA submission and launch preparation, with the possibility of being first to market.”
Pravin Dugel, president of Iveric Bio, added: “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.”
A breakthrough therapy designation is intended to accelerate the development and regulatory review of potential new medicines that aim to treat a serious condition and address a significant unmet medical need. The new medicine needs to show preliminary clinical evidence that the treatment may demonstrate substantial improvement in effectiveness over available therapies.