The European Commission approved avalglucosidase alfa, sold under the brand name Nexviadyme® by Sanofi, as a potential new standard of care for infantile-onset and late-onset Pompe disease. This makes avalglucosidase alfa a new treatment option for patients in Europe who have had Pompe disease for more than 15 years.

“The approval of Nexviadyme in Europe to treat Pompe disease is backed by a robust body of evidence showing clinically meaningful improvements that can impact quality of life,” said Benedikt Schoser, MD, senior consultant from the Friedrich-Baur-Institute, Department of Neurology at Ludwig-Maximilians-University in Munich, Germany.

“Nexviadyme’s demonstrated clinical benefit and molecular innovation bring a new treatment option to people living with Pompe disease who continue to face unmet needs.”


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Avalglucosidase alfa showed clinically meaningful improvements in key areas of disease burden in patients with Pompe disease including respiratory and motor function in a number of clinical trials.

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Avalglucosidase alfa is an enzyme replacement therapy that specifically targets the mannose-6-phosphate (M6P) receptor, which is the key player in the uptake of enzyme replacement therapies inside cells and in their transport to the lysosome.

Avalglucosidase alfa has a 15-fold higher level of M6P moieties on average compared to alglucosidase alfa, the only other enzyme replacement therapy available for Pompe disease. This means that the uptake of the therapy is higher, which could enhance glycogen clearance.

The treatment is already approved in the US, Japan, Canada, Switzerland, Australia, Brazil, Taiwan, and the UAE for the treatment of certain people with Pompe disease.

Pompe disease is caused by a mutation in the gene coding the acid alpha-glucosidase (GAA) enzyme, which is responsible for breaking down glycogen. The mutation results in GAA enzyme deficiency, which leads to a build-up of glycogen causing irreversible damage to skeletal and cardiac muscles.

Reference

Nexviadyme® (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe disease. News release. Sanofi; June 28, 2022.