Sanofi's avalglucosidase alfa shows meaningful results in late-onset Pompe disease

French pharma major Sanofi’s (Nasdaq: SAN) investigational enzyme replacement therapy (ERT), avalglucosidase alfa met the primary endpoint demonstrating non-inferiority in improving respiratory muscle strength, as measured by using percent-predicted forced vital capacity (FVC) in the upright position, compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease (LOPD).

The randomized, double-blind, Phase III, head-to-head COMET trial enrolled 100 previously untreated pediatric (≥ three years of age) and adult patients with LOPD across 56 centers in 20 countries. Patients were randomized to receive either avalglucosidase alfa 20mg/kg or alglucosidase alfa (standard of care) 20mg/kg intravenous infusion every two weeks for 49 weeks.

Patients treated with avalglucosidase alfa had a LS Mean 2.4-point difference in percent-predicted FVC compared to patients treated with standard of care (95% CI, -0.13 / 4.99) (p=0.0074) for non-inferiority.1