Using cipaglucosidase alfa/miglustat (cipa/mig) treatment for up to 104 weeks has a lasting impact on patients with late-onset Pompe disease (LOPD) and is well-tolerated, indicating that the treatment may offer long-term advantages, according to new results that will be presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
The phase 3 double-blind PROPEL study compared the effects of cipa/mig with alglucosidase alfa/placebo (alg/pbo) in adult ambulatory patients with LOPD over 52 weeks.
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The study also included an ongoing open-label extension (OLE) to evaluate the long-term safety and efficacy of cipa/mig. The outcomes of the study included various factors, including a 6-minute walk distance (6MWD), forced vital capacity (FVC), creatine kinase (CK), hexose tetrasaccharide (Hex4) levels, and safety. The data was reported for 104 weeks after the PROPEL baseline.
The results of the OLE phase of the study showed that 96.5% of patients previously treated with cipa/mig in PROPEL continued cipa/mig, while 97.4% of patients previously on alg/pbo switched to cipa/mig. Moreover, about 90.8% of patients remained in the OLE through the 52nd week with a 6MWD mean change of +3.1 for cipa/mig-cipa/mig and -0.5 for alg/pbo-cipa/mig in (enzyme replacement therapy) ERT-experienced patients and +8.6 for cipa/mig-cipa/mig and +8.9 for alg/pbo-cipa/mig in ERT-naive patients.
In addition, the mean FVC change was found to be -0.6 for cipa/mig-cipa/mig and -3.8 for alg/pbo-cipa/mig in ERT-experienced patients and -4.8 and -3.1 in ERT-naive patients.
At the same time, the mean CK reduction for the ERT-experienced and ERT-naive patients was found to be -132.1 and -216.9 for cipa/mig-cipa/mig and -161.0 and -218.6 for alg/pbo-cipa/mig, respectively.
Finally, the mean Hex4 reduction for the ERT-experienced and ERT-naive patients was found to be -1.9 and -2.9 for cipa/mig-cipa/mig and -2.6 and -2.9 for alg/pbo-cipa/mig, respectively.
The study also found that treatment-emergent adverse events occurred in 98.8% of cipa/mig-cipa/mig patients and 97.3% of alg/pbo-cipa/mig patients during the PROPEL and through week 52 of the OLE. Furthermore, three patients discontinued the treatment due to infusion-associated reactions during the OLE; no new safety signals were found. The above findings suggest that cipa/mig treatment was well-tolerated and had long-term benefits for patients with LOPD.
Mozaffar, T, Bratkovic, Byrne, B, et al. Long-term efficacy and safety of cipaglucosidase alfa/miglustat in patients with Pompe disease: a phase III open-label extension study (ATB200-07). Poster presented at: 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX.