AAN 2023: AT-GAA motor function gains now seen for 4 years in Pompe

Treatment with AT-GAA, Amicus Therapeutics’ experimental two-part therapy for Pompe disease, has now been shown to improve motor function, and to stabilize or improve lung function, for up to four years in adults. That’s according to new analyses from the ongoing Amicus-sponsored Phase 1/2 ATB200-02 trial…

FDA Filing of AT-GAA for Late-onset Pompe Expected by Mid-Year

Amicus Therapeutics anticipates completing a rolling application by mid-year to seek approval of its investigational therapy AT-GAA for late-onset Pompe disease in the U.S. The announcement follows a pre-filing meeting with the U.S. Food and Drug Administration (FDA). The rolling biologics license application (BLA), initiated in…