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MedCity News Amicus’s path to unseat Sanofi in rare disease gets steeper after Phase 3 miss Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter. Shares1   Amicus Therapeutics aims to position its experimental Pompe disease treatment as the new standard of care, supplanting a therapy from Sanofi. But securing approval will require explaining to regulators why they should look past the treatment’s failure to achieve the main goal of a pivotal study. The main goal of the Phase 3 study testing the Amicus drug, AT-GAA, was to measure how much further, on average, patients could walk in a six-minute test after 52 weeks of treatment. Pompe patients have an inherited enzyme deficiency that leads to a progressive weakening of their muscles.

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