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.
Keywords: GAA deficiency; Pompe disease; cipaglucosidase alfa; enzyme replacement therapy; miglustat.
Safety of home treatment with cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials Why was the study done? Late-onset Pompe disease (LOPD) is a rare, inherited disorder caused by the lack of a working enzyme called acid alpha-glucosidase, typically found inside muscle cells. This results in a loss of muscle and lung function for people living with LOPD. Cipaglucosidase alfa (cipa) is an enzyme replacement therapy that aims to replace the missing enzyme. Cipa is used with miglustat (mig), an oral enzyme stabilizer that protects cipa against breakdown in the bloodstream and increases the amount available for uptake into muscle cells. Cipa is given by infusion into a vein, typically over 4 hours, every 2 weeks. Some studies in LOPD and related disorders have shown better patient satisfaction and quality of life when these infusions are given in a home environment. What did the researchers do? The team studied results from three clinical trials in LOPD to better understand the safety of giving cipa+mig to people at home. What did the researchers find? In these clinical trials, 65 of 151 study participants received at least one infusion in a home environment. Overall, 2024 out of 9185 total cipa+mig treatments were given at home. Participants who received cipa+mig at home and those who received cipa+mig in the clinic had similar rates of side effects related to receiving medication by infusion (infusion-associated reactions). The most frequent infusion-associated reactions were fever after home infusion and headache and fever in the clinic. Only two participants who had a home-based treatment experienced a serious infusion-associated reaction that was considered an important medical issue. What do the findings mean? This analysis supports the safety of cipa+mig treatment at home for adults with LOPD. Why are these findings important? Eligible patients may be able to safely receive infusions at home with a low risk of infusion-associated reactions. This could make cipa+mig treatment more comfortable and convenient, and reduce the burden of traveling to a healthcare facility for treatment.
© The Author(s), 2026.