MHRA approval of Pombiliti + Opfolda to treat late- onset Pompe disease in the United Kingdom
Amicus Therapeutics , announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom has granted marketing authorizations for Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for adults living with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency)
The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD.
About Pombiliti + Opfolda: Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. In clinical studies, Pombiliti + Opfolda was associated with demonstrated improvements in both musculoskeletal and respiratory measures.
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