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Text|Pharmaceutical Mission Hills
According to the latest announcement on the official website of the Center for Drug Evaluation (CDE) of the State Food and Drug Administration, Sanofi (Sanofi) declared a new class 1 drug avalglucosidase alfa, which has obtained the implied license for clinical trials, and plans to develop an enzyme replacement for patients with early-onset Pompe disease Treatment
.
This is an enzyme replacement therapy under development.
Screenshot source: CDE official website
Pompe disease is a progressive, debilitating muscle disease that impairs movement and respiratory function
.
The disease is caused by a genetic defect or dysfunction of acid alpha-glucosidase (GAA) in the lysosome
In order to reduce glycogen accumulation, GAA must be delivered to lysosomes in muscle cells
.
The key way to transport the GAA enzyme to the intracellular lysosome is the mannose-6-phosphate (M6P) receptor
Avalglucosidase alfa, developed by Sanofi, enhances the delivery of GAA to muscle cell lysosomes by targeting the M6P receptor
.
Studies have shown that compared with the standard treatment of α-glucosidase, the product has an approximately 15-fold increase in M6P content, which is designed to help improve cell uptake of the enzyme and enhance target glycogen clearance
In August 2021, the FDA has announced the approval of this enzyme replacement therapy (trade name: Nexviazyme) for the treatment of patients with late-onset Pompe disease who are 1 year old and older
.
The approval is based on the positive results obtained from the pivotal phase 3 clinical trial COMET.
Sanofi once stated in a press release that avalglucosidase alfa is a potential new standard treatment for patients with late-onset Pompe disease
.
We hope that this therapy will also be approved in China as soon as possible, bringing new treatment options to more Pompe disease patients
Reference materials:
[1] Center for Drug Evaluation (CDE) of China National Medical Products Administration.
[3] Kishnani PS, Attarian S, Borges JL, et al.
