Full Press Release Details
FDA approves Nexviazyme (avalglucosidase alfa-ngpt),
an important new treatment option for late-onset Pompe disease
PARIS August 6, 2021 - The U.S. Food and Drug
Administration (FDA) has approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease, 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. Nexviazyme has been shown in clinical trials to
provide patients with improvements in respiratory function and walking distance.
Pompe disease is a debilitating
and progressive condition that significantly inhibits mobility and breathing, said Bill Sibold, Executive Vice President of Sanofi Genzyme. 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
Pompe disease affects an estimated 3,500 people in the United States and can present as infantile-onset Pompe disease (IOPD),
the most severe form of Pompe disease with rapid onset in infancy, 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
Targeted delivery to clear glycogen in muscle cells
Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), which results in build-up of complex sugars (glycogen) in muscle cells throughout the body. The accumulation of glycogen leads to irreversible damage to the muscles, including the diaphragm that supports respiratory function and
skeletal muscles that affect mobility, functional endurance and breathing.
The key pathway to transport GAA enzyme into the lysosomes in the cell is through the M6P receptor.
Nexviazyme is specifically designed to target M6P to improve cellular enzyme uptake and enhance glycogen clearance in target tissues with an approximate 15-fold increase in M6P content compared to
alglucosidase alfa, the comparator arm in the pivotal study.
Nexviazyme demonstrated improvements in pivotal study
Nexviazyme has demonstrated improvements for people living with late-onset Pompe disease. In the pivotal Phase 3 trial (COMET), Nexviazyme showed
improvements in respiratory function and walking distance measures in people with LOPD and established its safety profile.
Nexviazyme is a new and exciting therapeutic option for people with late-onset Pompe disease, 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.
Results from the COMET study
comparing Nexviazyme to alglucosidase alfa in LOPD included:
Nexviazyme, a new ERT for late-onset Pompe disease
Nexviazyme is administered as a monotherapy ERT every two weeks. The recommended dose is based on body weight (20 mg/kg for LOPD patients 30 kg or 40 mg/kg for LOPD patients <30 kg) and is administered incrementally via intravenous infusion. Nexviazyme is expected to be available in the U.S. in
As part of our commitment to ensure treatment access and
affordability for innovative therapies, Sanofi has decided to price Nexviazyme the same as alglucosidase alfa, the only other FDA-approved therapy for the treatment
of Pompe disease and the comparator arm in the pivotal study. Sanofi s CareConnectPSS Patient Support Services (1-800-745-4447, Opt. 3) provides personalized support for people and their families impacted by Pompe disease, including patients
transitioning to Nexviazyme.
The FDA approval follows a priority review by the FDA, which is reserved for medicines that, if approved, would
represent significant improvements in safety or efficacy in treating serious conditions. Previously, Nexviazyme received FDA Breakthrough Therapy and Fast Track designations for the treatment of people with Pompe Disease. The European Medicines
Agency s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa. While Sanofi is pleased with the CHMP s recognition of the clinically meaningful improvements demonstrated in the
avalglucosidase alfa development program, the CHMP has also rendered an opinion that avalglucosidase alfa does not qualify as a New Active Substance (NAS). As a result, Sanofi has requested a re-examination of
the CHMP opinion in relation to the NAS conclusion. Sanofi also filed avalglucosidase alfa in Japan in January 2021. The safety and efficacy of avalglucosidase alfa has not been fully evaluated by any regulatory authority outside of the U.S.
Sanofi is dedicated to supporting people
through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases
and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation
into healthcare solutions around the globe.
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