Amicus scores another approval in the UK for Pompe disease therapy, expects FDA OK in Q3
The Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has granted a marketing authorization for Amicus Therapeutics’ combo therapy for late-onset Pompe disease, a rare inherited lysosomal disorder, the company announced Tuesday.
The drugs, marketed as Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat), have also been recommended as first-line as well as later lines of therapy for the disease by the National Institute for Health and Care Excellence (NICE). Pombiliti is a long-term enzyme replacement therapy while Opfolda is an enzyme stabilizer.
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