Amicus Therapeutics announced that the European Commission, EC, has granted approval for Pombiliti, a long-term enzyme replacement therapy, ERT, used in combination with miglustat for adults with late-onset Pompe disease, LOPD. The Company has submitted the previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. The Committee for Medicinal Products for Human Use, CHMP, opinion for miglustat is expected in the second quarter of 2023. "Late-onset Pompe disease is a rare, neuromuscular disorder that can have devastating consequences for patients and their families. The European Commission approval for Pombiliti is another major step towards bringing this much needed, new treatment for all adults living in the EU with late-onset Pompe disease. It is the realization of the work of so many individuals and teams dedicated to the mission of improving the lives of people living with Pompe disease," said John F. Crowley, Executive Chairman of Amicus Therapeutics.
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