Amicus Therapeutics(US:FOLD) Globenewswire·2025-09-08 13:00Core Insights - The article presents new long-term data supporting the clinical profile of Pombiliti® + Opfolda® for treating late-onset Pompe disease (LOPD) [1][5][6] Group 1: Study Findings - The PROPEL open-label extension study showed improvements in muscle function, strength, and biomarkers over 208 weeks in ERT-experienced patients [2][4] - Key metrics included a +2.3% change in predicted six-minute walk distance (6MWD), +1.6 points in lower extremity manual muscle test (MMT) score, and a -160.0 reduction in serum creatine kinase (CK) levels [7][14] - No new safety signals were identified, with 41 patients experiencing treatment-related adverse events, leading to four discontinuations [4][6] Group 2: Treatment Indications - Pombiliti + Opfolda is indicated for adult patients with LOPD weighing ≥40 kg who are not improving on current enzyme replacement therapy (ERT) [3][16] - The treatment is not indicated for patients who are naïve to ERT [3] Group 3: Study Design - The PROPEL study was a 52-week, double-blind randomized global study involving 123 adult LOPD patients, assessing the efficacy and safety of cipaglucosidase alfa-atga + miglustat [9][10] - The open-label extension study included 118 patients, with 81 continuing treatment from the PROPEL study [12] Group 4: Company Overview - Amicus Therapeutics is focused on developing innovative therapies for rare diseases, with a commitment to improving the lives of patients with LOPD [19]