The US Food and Drug Administration (FDA) has granted Amicus' AT-GAA a Breakthrough Therapy Designation (BTD) for the treatment of late onset Pompe disease.

The drug 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 (rhGAA) enzyme with optimised carbohydrate structures, co-administered with AT2221, a pharmacological chaperone.

To receive BTD there must be preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint over available therapies, and the BTD for AT-GAA is based on clinical efficacy results from the ongoing ATB200-02 Phase I/II clinical study, including improvements in six-minute walk distance in late onset Pompe patients and comparison to natural history of treated patients.

John Crowley, chairman and chief executive of Amicus, stated, “There is an urgent need for new, second generation therapies for people living with lysosomal storage disorders, especially in a disease like Pompe. This important Breakthrough Therapy Designation from the FDA reflects the clinical data for our novel Pompe treatment paradigm AT-GAA.

“The BTD here also is based on the significant unmet need that remains for people living with Pompe disease, despite an approved therapy...This BTD, together with our results from the Phase II study and ongoing PROPEL pivotal study, support our strategy to advance AT-GAA as quickly as possible with the potential to become the new standard of care for all persons living with Pompe disease.”