Amicus Puts Positive Spin on Failure of its Phase III Trial for Pompe Disease

Amicus Therapeutics announced topline results from its Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease (LOPD). Although the results showed some positive data, it failed to hit the primary endpoint in a head-to-comparison with standard of care for Pompe disease. Company shares plunged 28% at the news.