Cipaglucosidase Alfa/Miglustat Therapy Improves Efficacy Outcomes in Enzyme Replacement Therapy-Experienced Patients With Late-Onset Pompe Disease
A new analysis of the phase 3 PROPEL study showed that switching to cipaglucosidase alfa/miglustat therapy enhanced outcomes among patients with late-onset Pompe disease.
A new effect size analysis of the phase 3, randomized, double-blind PROPEL study (NCT03729362) showed that enzyme replacement therapy (ERT)-experienced patients with late-onset Pompe disease (LOPD) who switched to Amicus Therapeutics’ 2-component therapy of cipaglucosidase alfa-atga (Pombiliti)/miglustat (Opfold) (cipa/mig) from alglucosidase alfa/placebo (alg/pbo) demonstrated improvements in various efficacy outcomes. Overall, these findings underscored the potential of this FDA-approved treatment as an option for this specific patient population.1
Presented at the
The PROPEL study compared the efficacy and safety of cipa/mig with alg/pbo among adults with LOPD; prior to study entry, 77% of the participants had previously received ERT with alg/pbo (median, 7.4 years). In this analysis, investigators aimed to explore the effect sizes of cipa/mig and alg/pbo for efficacy outcomes among ERT-experienced patients from PROPEL. Researchers calculated standardized within-group effect sizes (Cohen’s d for correlated measurements, baseline to week 52) for outcomes such as motor and lung function, muscle strength, quality of life and biomarkers in ERT-experienced patients by dividing mean change from baseline by standard deviations of the difference.
Further findings showed that patients who remained on alg/pbo had statistically significant within-group worsening for sitting and supine forced vital capacity; slow vital capacity; maximal expiratory pressure; creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and significant improvement only for Patient-Reported Outcomes Measurement Information System (PROMIS)–Dyspnea score. Notably, those who switched to cipa/mig did not show significant within-group worsening and demonstrated significant improvements for 6-minute walk distance; manual muscle tests; PROMIS–Fatigue score; Physician and Subject Global Impression of Change (5 subdomains); and CK and Hex4.
In September 2023, the
Following the announcement of the approval,
REFERENCES
1. Dimachkie MM, Bratkovic D, Byrne B, et al. Effect Size Analysis Of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa In Enzyme Replacement Therapy-Experienced Adults With Late-Onset Pompe Disease In PROPEL. Presented at: 2024 AANEM; October 15-18; Savannah, Georgia. Abstract 157.
2. Amicus Therapeutics announces FDA approval and launch of new treatment for Pompe disease. News release. September 28, 2023. Accessed October 15, 2024. https://ir.amicusrx.com/news-releases/news-release-details/amicus-therapeutics-announces-fda-approval-and-launch-new
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