FDA approves Amicus' Pombiliti + Opfolda combo therapy for Pompe disease
The FDA on Thursday approved Amicus Therapeutics’ combo therapy for a rare inherited lysosomal disorder called Pompe disease following several regulatory delays.
The drugs are marketed as Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat). Pombiliti is a long-term enzyme replacement therapy and Opfolda is an enzyme stabilizer. The therapy is already approved in the EU, and the UK’s National Institute for Health and Care Excellence recommended it last month as a first-line therapy as well as in later lines of treatment after it received approval there.
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