FDA Approves Avalglucosidase Alfa for Pompe Disease

The Food and Drug Administration (FDA) approved avalglucosidase alfa (avalGA) (Nexviazyme; Sanofi, Bridgewater, NJ) for the treatment of individuals age 1 year  and up with late-onset Pompe disease (LOPD). Treatment of people with LOPD with avalGA improved respiratory function and timed walking distance as well as the previously approved alglucosidase alfa (alGA)(Lumizyme, Sanofi). 

In the COMET study (NCT03019406), participants treated with avalGA had a 2.9±0.9 point improvement  in forced vital capacity (FVC) percent-predicted at week 49. This improvement was not statistically different from that seen with alGA meeting the measurement of noninferiority (P=.0074; 95% CI, -0.13, 4.99).  Participants treated with avalGA walked 32.2±9.9 meters farther at week 49 than at baseline.
 
“Nexviazyme is a new and exciting therapeutic option for people with LOPD,” said Mazen M. Dimachkie, MD, FAAN, FANA, professor of Neurology, chief of the Neuromuscular Division and executive vice chair of the Department of Neurology at the University of Kansas Medical Center. “The phase 3 study results showed meaningful improvements in respiratory function and walking distance, which are impactful in this serious condition.”
 
During the double-blind active-controlled period of 49 weeks, serious adverse reactions were reported in 2 (2%) participants treated with avalGA and in 3 (6%) participants treated with alGA. The most frequently reported adverse reactions (>5%) in avalGA-treated participants were fatigue, headache, hives, itching sensation, and nausea. Infusion-associated reactions were mild to moderate, including diarrhea, headache, hives, itching, and rash. There were no severe infusion-associated reactions.

Compared with alGA, avalGA has a 15-fold greater effect on enhancing glycogen clearance, the pathogenic step in Pompe disease.