On Friday, the U.S. Food and Drug Administration extended its review of an experimental combination medication developed by Amicus Therapeutics Inc. to treat Pompe disease, a muscle disorder, for a second time.

Pompe disease is a rare hereditary disorder caused due to the body′s inability to break down the complex sugar glycogen, which produces a buildup of the substance and impairs muscle function. In young patients, the disease may also result in heart failure.

In a regulatory document made last month, Amicus stated that an inspection of its partner WuXi Biologics′ Chinese production facility was not planned at the time.

The U.S. FDA had prolonged its review of both components of the combination therapy in May, stating that it needed more time to review the data provided by the firm. The organization attributed the delay to travel restrictions brought on by the epidemic, which prevented it from undertaking the necessary manufacturing site inspection before the decision was due.

Amicus needed approval from the FDA to develop the new therapy for Pompe disease AT-GAA using its drug migulstat in combination with the protein cipaglucosidase alfa that was produced in-house. The prolongation, according to analysts from BTIG and Needham & Co, will probably cause a delay in the launch of the combination therapy, AT-GAA by a quarter.

(Source: https://health.economictimes.indiatimes.com/news/pharma/fda-extends-review-of-amicus-muscle-disorder-therapy-for-second-time/95159020)