AT-GAA is an experimental therapy that is not currently approved for Pompe. It is being tested in ongoing clinical trials across the globe. The two-component treatment is designed to deliver to the body’s cells a functional version of the enzyme whose defect causes Pompe.
Each of the two components in AT-GAA is being considered by the U.S. Food and Drug Administration in separate applications. The FDA had initially set target dates in May for miglustat, and in July for cipaglucosidase alfa, but the agency has since extended its review. A decision on miglustat is expected by Aug. 29, while a decision on cipaglucosidase alfa should be made by Oct. 29.
Clinical trials testing AT-GAA have excluded people who are pregnant or breastfeeding, and have required that participants take measures to avoid conceiving a child while they are taking the experimental medication. Consequently, AT-GAA’s safety profile during pregnancy is unknown.
Some people with Pompe disease have seen results as soon as six months following treatment initiation. In the ATB200-02 study, improvements in six-minute walk distance were noted after half a year on AT-GAA, with patients walking a mean of more than 20 meters (66 feet) longer than they had when they started treatment.
Neither hair loss nor weight gain has been reported as common side effects of AT-GAA in clinical trials. Patients who experience unexpected changes after starting a new medication are advised to talk to their healthcare providers.
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