Posted by Michael Wonder on 25 Feb 2019
U.S. FDA grants breakthrough therapy designation to Amicus’ AT-GAA in late onset Pompe disease
25 February 2019 - First breakthrough therapy for an investigational treatment in Pompe disease.
Amicus Therapeutics today announced that the U.S. FDA has granted to Amicus a Breakthrough Therapy Designation (“BTD”) to AT-GAA for the treatment of late onset Pompe disease, an inherited lysosomal storage disorder caused by the deficiency of an enzyme known as acid alpha-glucosidase (GAA). AT-GAA is the first ever investigational product for Pompe disease to receive BTD. AT-GAA is a novel treatment paradigm consisting of ATB200, a unique recombinant human acid alpha-glucosidase enzyme with optimised carbohydrate structures, co-administered with AT2221, a pharmacological chaperone.
The BTD for AT-GAA is based on clinical efficacy results from the ongoing ATB200-02 Phase 1/2 clinical study, including improvements in six-minute walk distance in late onset Pompe patients and comparison to natural history of treated patients.
