Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials.

BACKGROUND

Late-onset Pompe disease (LOPD) is caused by a deficiency of the acid α-glucosidase enzyme. In LOPD treatment, enzyme replacement therapy is delivered via intravenous infusion, typically in clinical settings. Cipaglucosidase alfa is delivered with the oral enzyme stabilizer miglustat (cipa + mig).

OBJECTIVES

Evaluate the safety of cipa + mig home infusions.

DESIGN

Post hoc analysis of pooled safety data from three clinical trials in adults with LOPD (NCT02675465, NCT03729362, NCT04138277).

METHODS

The frequency and severity of infusion-associated reactions (IARs) during cipa + mig home and clinic administration were analyzed.

RESULTS

In total, 65/151 patients (43.0%) received ⩾1 cipa + mig home treatment. Of 9185 treatments, 2024 (22.0%) were administered at home. IAR frequency was similar for home (1.3%, 26/2024) and clinic (1.8%, 129/7161). The most frequent IAR following home infusion was pyrexia (6.2% of patients). Two patients with ⩾1 home-based treatment experienced serious IARs.

CONCLUSION

Analyses support the safety of home cipa + mig treatment in eligible adults with LOPD.