Home > Neurology > EAN 2022 > Miscellaneous > Long-term effects of avalglucosidase alfa in late-onset Pompe disease

Long-term effects of avalglucosidase alfa in late-onset Pompe disease

Presented By
Prof. Benedikt Schoser, Friedrich Baur Institute, Germany
Conference
EAN 2022
Trial
Phase 3, COMET
Doi
https://doi.org/10.55788/2a9dffa6
Patients with late-onset Pompe disease treated with avalglucosidase alfa (AVAL) for 97 weeks maintained improvement from baseline in respiratory function, motor function, muscle strength, and health-related quality-of-life in the extended treatment period of the COMET trial. Participants who switched to AVAL after 49 weeks of treatment with alglucosidase alfa (ALGLU) showed stabilisation of treatment effect. AVAL is a recombinant human GAA enzyme replacement therapy enriched with mannose-6-phosphate to increase cellular uptake compared with ALGLU. In the primary analysis period of the phase 3 COMET trial (NCT02782741), AVAL resulted in greater improvements in forced vital capacity (FVC), which was the primary endpoint, as well as in other outcomes, and a more favourable safety profile compared with ALGLU in 100 treatment-naïve patients with late-onset Pom...


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