Amicus Therapeutics has announced that the first patient has been dosed in their Phase 3 study of AT-GAA for patients with Pompe disease. The PROPEL Study is a 52 week double-blind clinical trial of 100 late-onset patients that studies the efficacy, safety and tolerability of AT-GAA. This furthers work that has been done to date in the Phase 1/2 study. AT-GAA is compared to the current standard of care, alglucosidase alfa, which is administered by bi-weekly infusions.

Both ERT-experienced and ERT-naive patients will be enrolled in as many as 90 locations globally. All patients that receive AT-GAA in this randomized clinical trial will receive the drug manufactured at the 1000L scale. This scale is intended for commercial and clinical use. Amicus also intends to start a smaller pediatric trial in 2019.

AT-GAA consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme, co-administered with AT2221, a pharmacological chaperone.


Tagged: chaperone, clinical trials, ERT