The FDA approval is based on positive Phase 3 data showing improvements in key disease burden measures and establishing its safety profile
FDA Centre for Drug Evaluation and Research. (Credit: The U.S. Food and Drug Administration/Wikipedia.)
Sanofi has received the US Food and Drug Administration (FDA) approval for Nexviazyme (avalglucosidase alfa-ngpt) to treat a type of Pompe disease.
Nexviazyme is an enzyme replacement therapy (ERT) designed to specifically target the mannose-6-phosphate (M6P) receptor, an important pathway for cellular uptake of ERT.
The US regulator indicated the drug as a monotherapy ERT administered every two weeks for the treatment of late-onset Pompe disease in patients aged one year and above.
The FDA approval of Nexviazyme follows a priority review, Breakthrough Therapy and Fast Track designations from the FDA to treat people with Pompe Disease.
Also, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has announced a positive opinion for the drug.
Sanofi Genzyme executive vice president Bill Sibold 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 rare diseases.”
Pompe disease is caused by a genetic deficiency or dysfunction that results in the accumulation of complex sugars (glycogen) in muscle cells throughout the body.
The accumulated glycogen causes irreversible damage to the muscles, including the diaphragm that supports respiration and skeletal muscles that affect mobility.
Pompe disease may progress into a most severe form called infantile-onset Pompe disease (IOPD) in infants, and into late-onset Pompe disease (LOPD) in all age groups.
People with LOPD may require mechanical ventilation for breathing assistance or a wheelchair for mobility, as the disease progresses.
The US FDA approval is based on positive results from Phase 3 COMET trial, in which Nexviazyme demonstrated improvements for people living with late-onset Pompe disease.
In the Phase 3 trial, Nexviazyme showed improvements in respiratory function and walking distance measures in people with LOPD, and its safety profile was established.
University of Kansas Medical Centre professor of neurology Mazen M Dimachkie 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.”
Last week, Sanofi has agreed to acquire clinical-stage mRNA therapeutics company Translate Bio for a total equity value of around $3.2bn.