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EU health regulator backs Amicus therapy for muscle disorder
2022-12-16
上市批准
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.
(This story has been corrected to say 2023, not 2022, in the last paragraph)
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