The U.S. Food and Drug Administration (FDA) has approved the combination of cipaglucosidase alfa-atga plus miglustat (Pombiliti+ Opfolda) to treat adults with late-onset Pompe disease. More specifically, the treatment is approved to treat those adults weighing more than 40 kg who are currently not improving with their current enzyme replacement therapy (ERT).
Late-onset Pompe disease is a rare, threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of glycogen that leads to muscle damage.
Cipaglucosidase is a recombinant human GAA enzyme (rhGAA). Miglustat stabilizes the enzyme in the blood.
The FDA reviewed data from the Phase 3 pivotal study (PROPEL). That data was published in Lancet Neurology and showed the oral therapy to be slightly superior to ERT in the six minute walk test, although that difference was not shown to be statistically significant. It is speculated that other data was presented to the FDA to show the benefits of an oral therapy for those not improving with ERT.
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Reference
Schoser B et al. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Lancet Neurol 2021; 20: 1027-1037.