US FDA approves Amicus Therapeutics' muscle disorder treatment (updated)

Amicus Therapeutics

28/09/2023 16:08

(Adds details on therapy in paragraph 3, background on disease
in paragraph 4)
       Sept 28 (Reuters) - Amicus Therapeutics  FOLD.O  said on
Thursday the U.S. Food and Drug Administration (FDA) had
approved its therapy to treat a muscle disorder called Pompe
disease.
    The approval comes after a long delay in bringing the
therapy to market. The health regulator last October extended
its review for a second time following delays in inspections
caused by pandemic-related travel restrictions.
    The company was seeking the health regulator's nod for the
expanded use of its therapy, AT-GAA, which is combination of
drug migulstat and lab-made protein cipaglucosidase alfa.
    The therapy is indicated for use in adults living with
late-onset Pompe disease and those without an improvement on
their current enzyme replacement therapy, the company said.
    Pompe disease is a rare genetic condition in which the body
is unable to break down the complex sugar glycogen, leading to a
buildup of the same, which causes muscular impairment. The
condition may also lead to heart failure in young patients.
    

 (Reporting by Sriparna Roy in Bengaluru; Editing by Shinjini
Ganguli)
 ((Sriparna.Roy@thomsonreuters.com;))