Avalglucosidase alfa (Nexviazyme®) stabilized or improved motor function in both patients with infantile- and late-onset Pompe disease, according to the long-term results of the phase 3 COMET (NCT02782741) and phase 2 mini-COMET (NCT03019406) trials that were presented at the 18th Annual WORLDSymposium 2022.

Patients enrolled in the long-term extension of the COMET study showed a 2.65 (1.05) point improvement compared to baseline in forced vital capacity percent-predicted if they received avalglucosidase alfa during the entire trial (97 weeks). Those who received alglucosidase alfa during the initial 49-week trial and avalglucosidase alfa during the extension period showed a 0.36 (1.12) point improvement compared to baseline.

The patients who received avalglucosidase alfa during the full study also increased their 6-Minute Walk Test score by 18.6 (12.01) meters compared to baseline, while the patients receiving avalglucosidase alfa only during the extension had an average increase of 4.56 (12.44) meters compared to baseline.

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During the long-term extension of the mini-COMET trial, the patients showed stable or improved motor function on the Gross Motor Function Measure, Quick Motor Function Test total percent score, and Pompe-Pediatric Evaluation of Disability Index Functional Skills Scale at week 97. All patients also had normal values on their left ventricle mass z-score.

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“These new data demonstrate the impact of avalglucosidase alfa to provide sustained effect in key disease measures for both late-onset and infantile-onset Pompe disease. The research also adds to a growing body of evidence supporting this treatment’s use as a new standard of care for this debilitating, life-long disease,” Alaa Hamad, MD, MPH, MBA, global head of Medical Affairs Rare Diseases at Sanofi said.

At the conference, the trial design of the new Baby-COMET (NCT04910776) study was discussed. The trial is a phase 3, single group, open-label, multicenter study which will recruit treatment-naïve patients with infantile-onset Pompe disease 6 months of age or less to investigate the effects of avalglucosidase alfa on overall and ventilator-free survival.

A total of 95 patients entered the extension portion of COMET and 86 remained at the last follow-up. No new safety signals were observed during the extension period and 5 patients discontinued treatment due to adverse events. Serious treatment-emergent adverse reactions potentially related to treatment were observed in 6 patients.

All 22 patients from the mini-COMET study continued into the extension study and were given up to 40 mg/kg doses of avalglucosidase alfa regardless of whether they received alglucosidase alfa during the initial portion of the trial. No serious or severe treatment-related adverse events were reported.


Nexviazyme® (avalglucosidase alfa) shows sustained improvements in respiratory function and mobility in patients with Pompe disease. News Release. Sanofi; February 8, 2022.