Astellas Submits NDA for Conditional Approval of Avacincaptad Pegol for Geographic Atrophy in Japan

Astellas Pharma Inc. announced the submission of a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for Conditional Approval of avacincaptad pegol intravitreal solution (ACP), a synthetic aptamer that inhibits the complement C5 protein, for the treatment of GA secondary to AMD. If approved, ACP has the potential to become the first and only GA treatment available in Japan.

GA is a progressive form of AMD that can cause irreversible vision loss, with no treatments currently approved outside the US or Australia. Globally, over five million people are estimated to have GA and, without timely treatment, an estimated 66% of people living with GA may become legally blind or severely visually impaired. As a result, GA secondary to AMD has a substantial impact on patients’ daily lives and psychological wellbeing.

Marci English, Vice President, Head of BioPharma and Ophthalmology Development, Astellas
“Today’s submission comes as good news to people in Japan living with geographic atrophy who have no approved treatment options for this devastating disease. If approved, avacincaptad pegol has the potential to be the first and only treatment to slow disease progression for eligible patients in Japan. As such, we are committed to working with regulatory authorities in Japan to ensure that patients can benefit from this vital new treatment.”

The NDA submission is based on results of overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled clinical trials, which evaluated the safety and efficacy of monthly 2mg intravitreal administration of ACP in patients with GA secondary to AMD.⁶ The data from both trials demonstrates that ACP slows GA lesion growth and has a favorable safety profile. Sustained efficacy of ACP, as shown in the restriction of lesion growth over time, was observed over a follow up of two years in GATHER1 and GATHER2 studies.

This submission will have no impact on the financial forecasts of the current fiscal year ending March 31, 2025.