[Pubmed] Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from

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Ther Adv Rare Dis. 2026 Jan 31;7:26330040261416943. doi: 10.1177/26330040261416943. eCollection 2026 Jan-Dec.

ABSTRACT

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.

PMID:41631150 | PMC:PMC12861377 | DOI:10.1177/26330040261416943


Source: https://pubmed.ncbi.nlm.nih.gov/4163115 ... 22+67771e2