FDA Extends PDUFA Dates for Amicus AT-GAA NDA, BLA

FDA has extended by 90 days its review period for an Amicus Therapeutics BLA for cipaglucosidase alfa and its NDA for miglustat, the two components in the company’s AT-GAA, intended for late-onset Pompe disease. The new PDUFA action dates for the BLA and NDA are 8/29 and 10/29, respectively.

The action date extensions are to give the agency additional time to review information submitted by Amicus as part of the review process, a company statement says. The extension was not related to requests for any additional clinical data, it says.

AT-GAA is described as an investigational two-component therapy that in preclinical studies was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect, and improvements in muscle strength. FDA has granted AT-GAA breakthrough therapy designation.