Sanofi Pompe bay disease therapy is eligible for FDA priority review
Last Update: 2021-03-05
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, Sanofi announced that the U.S. FDA has accepted the company's Biological Products Licensing Application (BLA) for avalglucosidase alfa for the treatment of glycogen accumulation disease (I. , II. type) (aka Pompeid disease, Pompeid disease) patients as a long-term enzyme replacement therapy. The FDA also granted priority review of the application, which is expected to be answered by May 8 next year.
Pompeia is a rare defying muscle disease that affects an individual's mobility and breathing ability. In the United States, an estimated 3,500 people suffer from the disease, which can be developed at any age, from inferfested to adulthood. It is caused by genetic defects or dysfunctions of acidic α-glucosidease (GAA) in lysosomes, resulting in the accumulation of glycogen in muscle cells throughout the body, which eventually leads to irreversible muscle damage, including respiratory and shin muscles that support lung function, and other skeletal muscles that affect the ability to function.
Avalglucosidase alfa is an enzyme replacement therapy designed to improve GAA delivery to muscle cells. To reduce glycogen accumulation, GAA must be delivered to lysosomes in muscle cells. Sanofi's research focused on enhancing GAA delivery to muscle cell lysosomes by targeting glyceride-6-phosphoric acid (M6P) subjects that play a key role in GAA transport. Compared to the standard treatment α-glucosidease, the M6P content of avalglucosidase alfa is increased approximately 15 times to help improve cell intake of enzymes and enhance target glycogen removal. This in-study therapy has been approved by the FDA as a breakthrough therapy and fast-track qualification for the treatment of Pompeia. If approved, a potential new standard of treatment will be provided for these patients.
This BLA is based on the results of two clinical trials. A key double-blind drug-controlled Phase 3 clinical trial assessed the safety and efficacy of avalglucosidase alfa compared to α-glucosidease (standard treatment) in patients with late-haired Pompeidase. Another Phase 2 clinical trial evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infant seizures who had previously been treated with α-glucosidease. The results of both clinical trials were presented at a medical conference earlier this year. (Drug Mingkangde)
. . . FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease. Retrieved November 17, 2020, from
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