EU health regulator backs Amicus therapy for muscle disorder (updated)

Amicus Therapeutics

16/12/2022 13:56

(Adds background on disease, FDA extension)
       Dec 16 (Reuters) - The European Union's health regulator
said on Friday it had recommended granting marketing
authorization for Amicus Therapeutics Inc's  FOLD.O 
experimental therapy to treat Pompe disease, a muscle disorder.
    Pompe disease is a rare genetic condition that leads to a
buildup of the complex sugar glycogen, causing muscular
impairment. The condition may also lead to heart failure in
young patients. 
    The European Medicines Agency's decision comes amid delays
for the drug in the United States.
    The U.S. Food and Drug Administration (FDA) in October had
extended the review of the drug for the second time, blaming the
delay on pandemic-related travel restrictions that kept it from
conducting a required inspection of the manufacturing site.
    The EU agency's decision only applies to Amicus' lab-made
protein cipaglucosidase alfa, which is used in combination with
another drug called miglustat.
    Amicus expects a recommendation on miglustat in the second
quarter of 2022. 
 (Reporting by Khushi Mandowara and Sriparna Roy; Editing by
Krishna Chandra Eluri)
 ((Khushi.Mandowara@thomsonreuters.com;))