FDA delays Amicus Therapeutics' Pompe disease drug review

Nov. 1, 2022

The FDA has pushed back the review of Amicus Therapeutics’ drug for Pompe disease, due to ongoing COVID-19 travel restrictions that have kept the agency from being able to carry out its facility inspections. 

In the announcement shared last Friday, the company said that they remain confident that it's not a matter of ‘if’ but ‘when’ the investigational drug will be approved. The therapy, called AT-GAA, is meant to treat the inherited muscle disorder by delivering a recombinant version of an enzyme that is usually deficient in Pompe patients.

The therapy delivers a functional copy of the GAA or enzyme acid alpha-glucosidase, administered with the intention to improve its uptake into cells. 

The FDA had previously granted Breakthrough Therapy Designation to the drug based on phase 1/2 clinical study results. Pompe disease is a rare, often fatal genetic disorder, that disables the heart and skeletal muscles.