Japan-based major Astellas Pharma (TYO: 4503) announced that it has submitted a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) seeking conditional approval for its avacincaptad pegol intravitreal solution (ACP). The drug is a synthetic aptamer that inhibits the complement C5 protein and is intended for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Acquisition and Global Approval Landscape
Astellas acquired ACP through its SUD5.9 billion acquisition of US biotech Iveric Bio. The drug was first approved in the US in August 2023 under the trade name Izervay for the treatment of GA secondary to AMD. Geographic atrophy is a progressive form of AMD that can cause irreversible vision loss. Currently, no treatments are approved outside the US or Australia for GA. Globally, over five million people are estimated to have GA, with approximately 66% of patients at risk of becoming legally blind or severely visually impaired without timely treatment.
Clinical Trial Results and Regulatory Submission
The NDA submission leveraged results from overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled trials. These studies assessed the safety and efficacy of monthly 2 mg intravitreal administration of ACP in patients with GA secondary to AMD. Data from both trials demonstrated that ACP slows GA lesion growth and has a favorable safety profile.-Fineline Info & Tech
