FDA approves Nexviazyme as treatment for late-onset Pompe disease
pharmafile | August 9, 2021 | News story | Sales and Marketing |
The FDA has approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease.
Pompe disease is a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.
Nexviazyme is an enzyme replacement therapy (ERT) designed to specifically target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of enzyme replacement therapy in Pompe disease.
In clinical trias, Nexviazyme has been shown to provide patients with improvements in respiratory function and walking distance.
Bill Sibold, Executive Vice President of Sanofi Genzyme, said: “Pompe disease is a debilitating and progressive condition that significantly inhibits mobility and 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.”
Mazen M Dimachkie, Professor of Neurology, Chief of the Neuromuscular Division and Executive Vice Chair of the Department of Neurology at the University of Kansas Medical Center, said: “Nexviazyme is a new and exciting therapeutic option for people with late-onset Pompe disease.
“The Phase 3 study results showed meaningful improvements in respiratory function and walking distance, which are impactful in this serious condition.”
Pompe disease affects an estimated 3,500 people in the United States and can present as infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD), which progressively damages muscles over time.
LOPD symptoms may present at any age. However, due to the wide spectrum of clinical presentations and progressive nature of the disease, it can take seven to nine years before patients receive an accurate diagnosis. As the disease progresses, people with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility.
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