FDA approves Sanofi’s Nexviazyme for late-onset Pompe disease

The US Food and Drug Administration (FDA) has approved Sanofi’s enzyme replacement therapy Nexviazyme for patients one year of age and older with late-onset Pompe disease (LOPD).

LOPD is a hereditary, progressive muscle disorder that impairs the ability to move and breathe. Nexviazyme targets the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of enzyme replacement therapy in Pompe disease. In the phase 3 COMET trial, LOPD patients treated with Nexviazyme showed improvements in respiratory function and walking distance.

Bill Sibold, executive vice president of Sanofi Genzyme, said: “For decades, we’ve made it our responsibility to research how to target the M6P receptor, the key pathway for cellular uptake of enzyme replacement therapy. Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases.”

“Nexviazyme is a new and exciting therapeutic option for people with late-onset Pompe disease,” said Mazen Dimachkie, professor of neurology, chief of the neuromuscular division and executive vice chair of the Department of Neurology at the University of Kansas Medical Centre. “The phase 3 study results showed meaningful improvements in respiratory function and walking distance, which are impactful in this serious condition.”

Late last year, Bayer established a new strategic platform focused on cell and gene therapy, with AskBio’s adeno-associated gene therapy platform reportedly showing promise in research programmes focused on Pompe disease, as well as Parkinson’s and congestive heart failure.