Amicus Therapeutics reported encouraging long-term data from their phase 1/2 study evaluating AT-GAA in enzyme replacement therapy (ERT)-naïve adults with Pompe disease.

The results, announced in a news release, include meaningful and lasting increases in the 6-Minute Walk Test (6MWT) distance and forced vital capacity (FVC), among other outcome measures, over 36 months.

“These latest long-term data from our Phase 1/2 study of AT-GAA continue to represent very meaningful and durable improvements in functional outcomes, as well as persistent reductions in key biomarkers of muscle damage and disease substrate out to three years,” John F. Crowley, chairman and chief executive officer of Amicus Therapeutics said.

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“The results have been shared with the global regulatory authorities in the US and EU as part of their ongoing reviews. Compared with what is known about the natural history of both untreated and ERT-experienced Pompe patients, these results give great hope that AT-GAA indeed has the potential to become the new global standard of care for people living with Pompe disease.”

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In addition to positive results in terms of the 6MWT and FVC, study patients also experienced improved arm and shoulder strength and reductions in biomarkers of muscle damage and disease substrate (glycogen) in cells.

AT-GAA is an investigational treatment currently under global regulatory review, consisting of combined therapy with cipaglucosidase alfa (ATB200) and optimized carbohydrate structures such as bis-phosphorylated mannose-6 phosphate glycans. It is delivered in conjunction with miglustat (AT2221), which stabilizes ATB200.

The open-label ATB200-02 study aimed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ATB200 over an 18-week study period. The study results were presented in Nashville, Tennessee at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference on March 16, 2022.


Amicus Therapeutics announces positive long-term data from phase 1/2 study of AT-GAA in Pompe disease at the 2022 MDA Clinical & Scientific Conference. News release. Amicus Therapeutics; March 16, 2022.