Avalglucosidase alfa appears to be more effective than alglucosidase alfa (ALG) in improving key aspects of Pompe disease (PD), which are relevant to the daily life of patients, according to a recently published posthoc analysis in Value in Health.

PD is a neuromuscular disease in which a deficit or absence of the lysosomal acid alpha-glucosidase enzyme (GAA) leads to lysosomal glycogen accumulation and impairment of muscular and cardiac tissue, the researchers noted. Enzyme replacement therapy represents the mainstay of PD therapy. ALG has been the preferred line of PD treatment for more than a decade, and although it has successfully increased survival rates and cardiac function, many symptoms remain, the authors added.

AVL has recently received European approval for the treatment of late-onset PD. Results from the COMET trial have suggested that ALV is superior to ALG in improving respiratory function, muscular endurance, and ambulation. 

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The authors aimed to perform a post hoc analysis of 3 patients’ reported outcomes (PROs) in the COMET trial, namely the Pompe Disease Symptom Scale (PDSS), Pompe Disease Impact Scale (PDIS), Rasch-Built Pompe-Specific Activity (R-PAct) scale. 

“The PDSS, PDIS, and R-PAct measure symptoms and functional limitations that are specific and important to PD,” the authors wrote. 

Researchers analyzed the percentage of multiPDSS domain responders, PDIS domains, as well as patients that switched from unable to do at baseline to able to do.

Results revealed AVL to be significantly superior to AGL after 49 weeks of treatment in multiPDSS domain responder analysis and PDIS domain. 

Similarly, significantly more patients receiving AVL were able to perform activities such as sitting, standing up, walking, bending the knee, and climbing stairs up and down in comparison to patients receiving ALG.

“These posthoc analyses extend the findings from exploratory endpoint analyses, showing positive, consistent trends throughout LOPD-specific PROs in favor of AVA over ALG on aspects of PD relevant to patients’ daily lives,” the authors wrote. 


Toscano A, van der Ploeg AT, Berger KI, et al. Avalglucosidase Alfa (AVA) improves symptoms and functioning in late-onset Pompe disease (LOPD) patients vs Alglucosidase Alfa (ALG): post-hoc analyses of patient-reported outcomes (pros) from Comet trial. Value Health. Published online December 01,2022. doi:10.1016/j.jval.2022.09.2005