Amicus Shares Encouraging Long-Term Data Of AT-GAA Study In Pompe Disease


Amicus Therapeutics Inc FOLD has announced additional results from a Phase 1/2 clinical study of AT-GAA in adult patients with Pompe disease.

  • Study participants treated with AT-GAA for up to 36 months showed persistent and durable effects on six-minute walk test (6MWT) distance and other measures of motor function and muscle strength, stability, or increases in forced vital capacity (FVC).
  • The data also exhibited persistent and durable reductions in key biomarkers of muscle damage and disease substrate across all patient cohorts.
  • AT-GAA is currently under global regulatory reviews. 
  • Also Read: Gene Therapy Player Amicus Therapeutics Terminates SPAC Merger, Shares Fall.
  • The FDA accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA, with action dates of July 29 and May 29
  • Long-term data from the Phase 3 PROPEL open-label extension study is expected to be presented later this year.
  • To date, adverse events have been generally mild and transient.
  • Price Action: FOLD shares are up 2.46% at $9.17 during the market session on the last check Wednesday.
Posted In: BiotechNewsHealth CareMoversTrading IdeasGeneralBriefsgene therapy