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

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.

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