FDA Defers Action On Amicus Therapeutics' Much Awaited Pompe Disease Therapy

The FDA deferred action on Amicus Therapeutics Inc's FOLD Biologics License Application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA for Pompe disease.
Due to restrictions on travel related to COVID-19, the FDA could not conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle.
The company continues to expect the FDA to approve the two components of AT-GAA, including the BLA and New Drug Application (NDA) for miglustat, together.
In May, the agency extended the review period for AT-GAA from August 29 to October 29.
While both applications remain under review, the FDA has not provided anticipated action date(s).
The company looks forward to a Committee for Medicinal Products for Human Use (CHMP) opinion in Europe by the end of the year.
AT-GAA is an investigational two-component therapy including cipaglucosidase alfa, a recombinant human acid alpha-glucosidase, to enhance uptake into cells, administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa.
Pompe disease is an inherited lysosomal disorder characterized by severe muscle weakness.
Price Action: FOLD shares closed 3.56% higher at $10.75 on Friday.