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EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy
PARIS October 2, 2020 The European Medicines
Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid -glucosidase deficiency).
Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.
Pompe disease is a rare, degenerative muscle disorder that can impact an individual s ability to move and breathe. It affects an estimated 50,000 people
worldwide and can manifest at any age from infancy to late adulthood.
The MAA is based on positive data from two trials:
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
respiratory muscles, such as the diaphragm muscle that supports the lungs, and other skeletal muscles that affect mobility. Avalglucosidase alfa is designed to improve the delivery of GAA enzyme into the lysosomes of muscle cells to breakdown
glycogen and help address respiratory impairment, as well as decreased muscle strength and function (i.e. mobility), which are critical manifestations of Pompe disease.
The Medicines and Healthcare Products Regulatory Agency in the UK has granted Promising Innovative Medicine designation for avalglucosidase alfa, an early
indication that the investigational therapy is a promising candidate for the Early Access to Medicines Scheme in the UK.i
The U.S. Food and Drug Administration has granted Breakthrough Therapy and Fast Track designations to avalglucosidase alfa.
Delivery of GAA to Clear Glycogen
To reduce the glycogen
accumulation caused by Pompe disease, the GAA enzyme must be delivered into the lysosomes within muscle cells. Research led by Sanofi has focused on ways to enhance the delivery of GAA into the lysosomes of muscle cells by targeting the mannose-6-phosphate (M6P) receptor that plays a key role in the transport of GAA.
Avalglucosidase alfa is designed with approximately 15-fold increase in M6P content, compared to alglucosidase alfa,
and aims to help improve cellular enzyme uptake and enhance glycogen clearance in target tissues.ii The clinical relevance of this difference has not been confirmed.
Avalglucosidase alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority worldwide.
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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| Media Relations Contact Sally Bain Tel.: +1 781 264 1091 sally.bain@sanofi.com | Sanofi Investor Relations Contacts Paris Eva Schaefer-Jansen Arnaud Delepine Yvonne Naughton Sanofi Investor Relations Contacts North America Felix Lauscher Fara Berkowitz Suzanne Greco IR main line: Tel.: +33 (0)1 53 77 45 45 ir@sanofi.com | |
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