Amicus Therapeutics, Inc. sees potential blockbuster sales for Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) by the end of the decade as the market for late-onset Pompe disease patients whose condition does not improve on enzyme replacement therapy (ERT) gradually expands, based on the forecast that the two-component therapy could claim more than half of that market.
The US Food and Drug Administration approved the combination of Pombiliti + Opfolda 28 September for late-onset Pompe disease (LOPD) patients weighing 40kg or more who are not improving on current ERT
Key Takeaways
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The FDA approved Pombiliti + Opfolda for late-onset Pompe disease in adults whose disease does not improve with enzyme replacement therapy
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