Sanofi has announced that the U.S. FDA approved its new treatment for children one year of age or older diagnosed with late-onset Pompe disease.
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Global biopharmaceutical firm Sanofi has announced that the U.S. Food and Drug Administration (FDA) approved its new treatment for children one year of age or older diagnosed with late-onset Pompe disease.
The new drug, Nexviazyme (avalglucosidase alfa-ngpt), is an enzyme replacement therapy (ERT) that specifically targets the mannose-6-phosphate (M6P) receptor which is the main pathway for cellular uptake of ERT in Pompe disease.
Pompe disease is a progressive and debilitating muscle disorder that impairs a person’s ability to breathe and move. It affects around 3,500 people in the U.S. alone. The disease presents itself either as an infantile-onset Pompe disease (IOPD), which is the most severe or as a late-onset Pompe disease (LOPD), which damages the muscles over time.
IOPD appears in infancy, while LOPD can occur at any age. However, because of the disease’s rapidly progressive nature, it usually takes as much as seven to nine years before a patient can receive an accurate diagnosis. As the illness progresses, those with LOPD might eventually need a wheelchair to be mobile or mechanical ventilation to continue breathing.
“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,” said Bill Sibold, Sanofi Genzyme’s executive vice president, in a press release.
Nexviazyme has been shown in clinical trials to provide patients with relief and improvements in walking distance and respiratory function. Pompe disease is caused by a genetic deficiency or dysfunction in the lysosomal enzyme acid alpha-glucosidase (GAA), which leads to a build-up of glycogen in muscle cells. The key pathway to transport GAA is the M6P receptor. Nexviazyme works by targeting M6P to improve cellular enzyme uptake and glycogen clearance.
The positive responses were observed in Sanofi’s Phase III COMET trial. Nexviazyme was given as monotherapy ERT every two weeks via intravenous infusion. The recommended dose is based on body weight. The drug is said to be available to the U.S. commercial market in the coming weeks.
“The Phase III study results showed meaningful improvements in respiratory function and walking distance, which are impactful in this serious condition,” said Mazen M. Dimachkie, M.D., FAAN, FANA, professor of neurology, Neuromuscular Division chief, and executive vice chairperson for the Neurology Department at the University of Kansas Medical Center.
Prior to the FDA’s approval, Nexviazyme received Breakthrough Therapy and Fast Track designations for patients with Pompe disease. Sanofi also filed avalglucosidase alfa in Japan in January 2021, but results have yet to be announced. The safety and efficacy of the drug have not been fully evaluated by any regulatory body outside the United States.
