Short of reaching high bar, Amicus’ Pompe treatment still headed for FDA review

Amicus still believes its two-component therapy can become a new standard of care in Pompe disease, but nearly a third of the company’s value has vaporized after it said the treatment missed the primary endpoint in the Phase III PROPEL study.

AT-GAA from Amicus Therapeutics Inc. (NASDAQ:FOLD) failed to significantly improve six-minute walk distance (6MWD) at 52 weeks compared with the current SOC, Lumizyme/Myozyme alglucosidase alfa, although Amicus’ therapy led to a numerically greater distance (20.8 meters vs. 7.2 meters, p=0.072). Sanofi (Euronext:SAN; NASDAQ:SNY) markets Lumizyme...