Cipaglucosidase/Miglustat for Pompe Disease: The Long-Term Safety, Efficacy

Among patients with late-onset Pompe disease, cipaglucosidase/miglustat is beneficial and well-tolerated at 104 weeks, according to study results presented at the 2023 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held from November 1 to 4 in Phoenix, Arizona. 

The findings of the PROPEL study (ClinicalTrials.gov Identifier: NCT03729362) supported the benefits of cipaglucosidase/miglustat vs enzyme replacement therapy (ERT) and placebo in the treatment of ambulatory patients with late-onset Pompe disease.

Researchers of the current open-label extension study (ClinicalTrial.gov Identifier: NCT04138277) aimed to determine the long-term safety and efficacy of cipaglucosidase/miglustat in patients with Pompe disease.

Study outcomes were 6-minute walk distance, forced vital capacity (FVC), creatine kinase, and hexose tetrasaccharide (Hex4) levels; and safety of cipaglucosidase/miglustat.

Changes from baseline in the PROPEL study to week 104 in the current study were recorded (Table).

Overall, 119 participants (91 ERT-experienced) from the PROPEL trial were enrolled in the current analysis. Of the total cohort, 82 participants continued to receive cipaglucosidase/miglustat and 39 who received placebo in the PROPEL trial were switched to cipaglucosidase/miglustat.

Table: Mean percentage changes (SD) in outcomes from the PROPEL to open-label trial.

ERT: enzyme replacement therapy; FVC: forced vital capacity

Both creatine kinase and Hex4 levels improved with cipaglucosidase/miglustat as well.

With regard to safety, 3 patients discontinued the study due to adverse reactions, including urticaria, hypotension, and anaphylaxis; however, no new safety signals were found.

Overall, the researchers concluded, “Data demonstrate treatment with cipa/mig [cipaglucosidase/miglustat] up to 104 weeks was associated with a durable effect and was well tolerated … [among] patients with LOPD [late-onset Pompe disease].”

Disclosure: This research was supported by Amicus Therapeutics, Inc.