Amicus Therapeutics Reports 4-Year Muscle Function And Biomarker Data From PROPEL Study Of Cipaglucosidase Alfa + Miglustat In Late-Onset Pompe Disease

Author: Benzinga Newsdesk | September 08, 2025 09:05am

Amicus Therapeutics (NASDAQ:FOLD), today announced the presentation of new 4-year muscle function, muscle strength and biomarker endpoints from the PROPEL open-label extension (OLE) study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan.

The new analysis, "208-week efficacy and safety of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers," was conducted on the 82 patients (62 ERT-experienced and 20 ERT-naïve) who were randomized to cipa+mig in PROPEL and who enrolled in the open label extension (OLE).

Pombiliti + Opfolda is indicated in the U.S. for the treatment of adult patients with LOPD weighing ≥40 kg and who are not improving on their current ERT. In the U.S., Pombiliti + Opfolda is not indicated for patients naïve to ERT treatment. The data summarized below are from the ERT-experienced group in the OLE. Pulmonary function data from the PROPEL OLE will be presented separately at an upcoming conference.

The mean age was 48.8 years and the median duration of prior ERT in the ERT-experienced cohort at PROPEL baseline was 7.5 years. In the ERT-experienced group, the measures of muscle function, muscle strength and biomarkers improved and/or were sustained out to 208 weeks. Mean change from PROPEL baseline to week 208 were as follows:

• Percent predicted six-minute walk distance (6MWD), +2.3%;
• Lower extremity manual muscle test (MMT) score, +1.6 points;
• Gait-Stairs-Gowers-Chair (GSGC) score, +0.1 points;
• PROMIS® physical function score, +1.2 points;
• Serum creatine kinase (CK) levels, -160.0 serum CK; and,
• Urine hexose tetrasaccharide (Hex4), -1.9 mmol/mol creatinine.

No new safety signals were identified. Up to week 208, 41 patients experienced treatment-related treatment-emergent adverse events (TEAEs), leading to four cipa+mig discontinuations, and two patients experienced serious treatment-related TEAEs, leading to two cipa+mig discontinuations. One patient experienced a TEAE (worsening of Lewy body disease) unrelated to study treatment, which led to death; the patient had discontinued from the study prior to death.

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