FDA lifts clinical hold on Astellas Pompe disease gene therapy trial

Jan. 20, 2023

Tokyo-based Astellas Pharma announced this week that the FDA has lifted the clinical hold on its phase 1/2 clinical trial evaluating its investigational late-onset Pompe disease drug, AT845.

The trial, called FORTIS, is evaluating the safety, tolerability and efficacy of the drug — a novel gene replacement therapy — in a multicenter, open-label, ascending dose, first-in-human study.

Pompe disease is a metabolic disorder that’s characterized by muscular degeneration and caused by mutations in the acid alpha-glucoside gene. 

Astellas developed AT845 to deliver a functional copy of this gene using an AAV8 vector that can be successfully transduced to provide therapeutic effects directly in the affected tissue. 

Back in June of 2022, the FDA placed the clinical hold on the trial after one of the participants suffered a serious adverse event of nerve damage, known as peripheral sensory neuropathy.