Sanofi's Pompe disease treatment has received FDA priority review and is expected to become a new standard treatment

Sanofi has 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. type, II. type) (also known as Pompeid disease, Pompeid disease) patients as a long-term enzyme replacement therapy.
fda also granted priority review of the application and is expected to respond by May 8 next year.
is a rare defying muscle disease that affects an individual's mobility and breathing ability.
the United States, an estimated 3,500 people suffer from the disease, from infested to adulthood, at any age.
It is caused by a genetic defect or dysfunction of the acidic α-glucosidease (GAA) in the lysosome, 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.
led by Sanofi focused on enhancing GAA's delivery to muscle cell lysosomes by targeting glycosaccharide-6-phosphate (M6P) subjects that play a key role in GAA transport.
the M6P content of avalglucosidase alfa increased by about 15 times compared to the standard treatment α-glucosidease, designed to help improve cell enzymatic intake and enhance target glycogen removal.
in-study therapy has been approved by the FDA as a breakthrough therapy and fast-track qualification for the treatment of Pompeia.
, if approved, will provide these patients with a potentially new standard of treatment.
deficiency in glycogen degradation in patients with Pompe disease (Photo: pompe.com) The BLA is based on the results of two clinical trials.
a key double-blind drug-controlled Phase 3 clinical trial to assess the safety and efficacy of avalglucosidase alfa compared to α-glucosidease (standard treatment) in patients with late-hair pompeidase.
phase 2 clinical trial evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infestion who had previously been treated with α-glucosidease.
results of two clinical trials were presented at a medical conference earlier this year.