FDA approves Pombiliti + Opfolda combination to treat late stage Pompe disease- Amicus Therapeutics
The FDA has approved Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) combination therapy for people with late-onset Pompe disease (LOPD) who weigh at least 40 kg and are not improving on current enzyme replacement therapy (ERT).
Pombiliti + Opfolda will be distributed and marketed in the United States by Amicus Therapeutics.
Pombiliti + Opfolda therapy should be started 2 weeks after the last ERT dose. Pombiliti has a recommended dosage of 20 mg/kg administered every other week as an intravenous (IV) infusion over 4 hours. Pombiliti therapy must be preceded by oral administration of Opfolda (available in 65 mg capsules) approximately 1 hour before IV infusion. Pombiliti’s label contains a boxed warning for hypersensitivity reactions including anaphylaxis, infusion-associated reactions, and risk of acute cardiorespiratory failure in susceptible patients.
150 mins
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.