Avalglucosidase alfa (ALV) appears to be more effective than alglucosidase alfa (ALG) in preventing the requirement for noninvasive ventilation and a wheelchair in patients with late-onset Pompe disease (PD), according to a recently published study in Value in Health.

Decreasing the progressive loss of muscular and respiratory function associated with late-onset PD in order to prevent the need for noninvasive ventilation or permanent disability requiring wheelchair use is currently one of the main objectives of enzyme replacement therapy in Pompe disease, the authors noted.

ALV a is a recombinant human GAA enzyme replacement therapy that targets the mannose-6-phosphate-receptor with the aim of increasing glycogen clearance. Results from the COMET trial suggested that ALV offered a clinical improvement over ALG, the current standard of care, the researchers added. 


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“But evaluating treatment effects on the need for ventilation and wheelchair support is difficult in a clinical trial due to the length of observation and large sample size required,” the authors wrote.

Therefore, researchers aimed to evaluate the impact of ALV on ventilation and wheelchair use through a simulation of treatment effects using data from clinical trials as well as observational data. The results from phase 2 and 3 clinical trials on ALV, data in the public domain, and data from the Pompe registry of ALG were used to build a disease model.

Moreover, the data was later implemented in a Discretely Integrated Condition Event (DICE) to predict the proportion of patients that would eventually require noninvasive ventilation or wheelchair use when receiving ALV and ALG, respectively. 

The simulation revealed that approximately 55% of patients receiving ALV would eventually require noninvasive ventilation compared with 70% of patients receiving ALG. Similarly, there was a reduction of approximately 20% in wheelchair use in patients receiving ALV. The time necessary for requiring ventilation and wheelchair use was reduced by 4.6 and 6.4 years, respectively.

“The findings from simulation suggest that the immediate benefits of AVA vs. ALG will lead to reduction in the need for assisted ventilation and wheelchair use in patients with LOPD,” the authors concluded.

Reference

Fournier M, Caro JJ, Coughlan A, et al. CO20 predicted time to wheelchair and ventilation events comparing avalglucosidase Alfa (AVA) versus (VS) alglucosidase Alfa (ALG) using a model of late-onset Pompe disease (LOPD). Value Health. Published online December 1, 2022. doi:10.1016/j.jval.2022.09.100