ATB200

Unassigned

New Medicines

Glycogen storage disease type II (Pompe disease) - first-line

Information

New molecular entity
Amicus Therapeutics
Amicus Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Enzyme replacement therapy. Highly targeted rhGAA (ATB200) is co-administered with a chaperone (AT2221).
The overall prevalence has been estimated at 1 in 40,000, with 1 in 138,000 for the infantile form and 1 in 57,000 for the adult form [1].
Glycogen storage disease type II (Pompe disease) - first-line
Parenteral