Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease. This threatment involves an enzyme replacement infusion of ATB200, coadministered with AT2221. ATB200 is a recombinant human acid alpha-glucosidase (rhGAA) enzyme. AT2221 is a pharmaceutical chaperone.

To receive BTD, the therapy must show significant improvement over available treatment in at least one end-point. At the recent 2019 WORLD Symposium, Amicus presented updated data on their clinical trials for AT2221/ATB200. It showed improvement in the six minute walk test (6MWT) over conventional treatment. Forced vital capacity was essentially unchanged when compared to conventional treatment.

BTD isn’t just a designation. It’s a program that includes features that should enable Amicus to fast track development and bring the therapy to market sooner.



Tagged: Breakthrough therapy designation, chaperone, clinical trials, ERT