Amicus' Pompe therapy approved in UK

US rare diseases focused biotech Amicus Therapeutics (Nasdaq: FOLD) today revealed that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorizations for Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for adults living with late-onset Pompe disease (acid α‑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.

In March 2023, Amicus announced that the European Commission had granted approval for Pombiliti (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). Miglustat is already approved as Galafold in Europe and the USA to treat Fabry disease, which like Pompe, is a glycogen storage disease.