FOLD - Amicus Therapeutics reports positive long term data of AT-GAA for Pompe disease

Amicus Therapeutics (NASDAQ:FOLD) reported additional results from a global phase 1/2 study (ATB200-02) of AT-GAA in adult patients with Pompe disease. Pompe disease is an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells. The company said patients treated with AT-GAA for up to 3 years 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), and reductions in biomarkers of muscle damage and disease substrate. The company added that safety profile aligned with previously reported data. To date, adverse events have been generally mild and transient. "The results have been shared with the global regulatory authorities in the US and EU as part of their ongoing reviews," said Amicus Chairman and CEO John Crowley. Long-term data from a phase 3 study, dubbed PROPEL, is expected to

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Amicus Therapeutics reports positive long term data of AT-GAA for Pompe disease