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2285 Efficacy and safety of avalglucosidase alfa in participants with late-onset pompe disease after 97 weeks of treatment during the COMET trial

作者

Robert Henderson, Priya S Kishnani, Jordi Díaz-Manera, Hani Kushlaf, Shafeeq Ladha, Tahseen Mozaffar, Volker Straub, Antonio Toscano, Ans T van der Ploeg, Kenneth I Berger, Paula R Clemens, Yin-Hsiu Chien, John W Day, Sergey Illarioshkin, Mark Roberts, Shahram Attarian, Gerson Carvalho, Young Chul Choi, Sevim Erdem-Özdamar, Ozlem Goker-Alpan, Anna Kostera-Pruszczyk, Kristina An Haack, Olivier Huynh-Ba, Swathi Tammireddy, Nathan Thibault, Tianyue Zhou, Mazen Dimachkie, Benedikt Schoser, COMET Investigator Group

发表日期

2022/8/1

来源

BMJ Neurology Open

卷号

期号

Suppl 1

出版商

BMJ Specialist Journals

简介

Objectives

Report efficacy/safety of avalglucosidase alfa (AVAL) in participants with late-onset Pompe disease in the extended treatment period (ETP) of Phase 3 COMET(NCT02782741) after a 49-week primary analysis period (PAP).

Methods

At PAP enrollment, participants were treatment-naïve (n=100; age 16–78 years). All 51 participants receiving AVAL 20mg/kg every 2 weeks (qow) in the PAP continued this in the ETP (AVAL-arm). Of 49 participants receiving alglucosidase alfa (ALGLU) 20mg/kg qow in the PAP, 44 entered the ETP switching to AVAL 20mg/kg qow (Switch-arm).

Results

Trends for improvement or stabilization from Baseline to Week 97 were observed for the primary and secondary outcomes of respiratory and motor function. Changes (LSmean[SE]) in forced vital capacity%predicted: AVAL-arm, +2.65[1.05]; Switch-arm, +0.36[1.12] and 6-minute walk test distance: AVAL-arm, +18.60[12.01]m …

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2285 Efficacy and safety of avalglucosidase alfa in participants with late-onset pompe disease after 97 weeks of treatment during the COMET trial

R Henderson, PS Kishnani, J Díaz-Manera, H Kushlaf… - 2022