Astellas Advances Geographic Atrophy Treatment In Japan Submits NDA

Astellas Pharma Inc. submitted a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for Conditional Approval of avacincaptad pegol intravitreal solution (ACP), which is a synthetic aptamer that blocks complement C5 protein for treating Geographic Atrophy secondary to AMD. The conditional approval of this drug could lead Japan to have its first medication specifically for GA treatment. 

Marci English, Vice President, Head of BioPharma and Ophthalmology Development, Astellas, said in a statement, “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.”

GA is a progressive AMD form that leads to permanent vision deterioration yet receives no external authorization besides the United States and Australia. The global GA patient population exceeds five million, and when left untreated, medical research predicts that 66% of people with GA will eventually develop severe vision impairments or blindness. The development of AMD secondary to GA produces substantial effects on the patient's daily activities and mental state.

The NDA submission is based on the results of overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled clinical trials, which evaluated the safety and efficacy of monthly 2 mg intravitreal administration of ACP in patients with GA secondary to AMD. The data from both trials demonstrate that ACP slows GA lesion growth and has a favorable safety profile. 6 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.