WuXi Biologics congratulates its strategic partner Amicus Therapeutics on receiving FDA Breakthrough Therapy Designation for AT-GAA (ATB200/AT2221) in 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 breakthrough designation.
The AT-GAA program was initiated at WuXi Biologics in 2012 with just an initial concept and now progresses through a pivotal study enabled by the global leading technology platform and unparalleled manufacturing capacity at WuXi Biologics. Just this month the two companies signed an exclusive manufacturing partnership, through which WuXi Biologics will provide dual commercial sources for both drug substance and drug product of ATB200 and will be the exclusive commercial drug substance partner and key commercial drug product supplier.
"We congratulate Amicus on achieving this exciting milestone," said Dr. Chris Chen, CEO of WuXi Biologics. "It is a great honor to enable global innovative partners like Amicus through our world-class development and manufacturing capacities and capabilities. We are confident of supplying this critical product to the global market and wish the program a great success to benefit patients worldwide."
This is the second product successfully receiving Breakthrough Therapy Designation that WuXi Biologics has enabled its partner to develop. Trogarzo, the first to do so, has been approved by the U.S. FDA in March 2018, marking the first commercial product manufactured by WuXi Biologics for the U.S. market.
AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, AT-GAA was associated with increased tissue enzyme levels, reduced glycogen levels in muscle, and improvements in muscle strength. A global Phase 1/2 study (ATB200-02) is ongoing to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA.