Sanofi’s (NASDAQ: SNY) next-generation enzyme replacement therapy (ERT), Nexviazyme (avalglucosidase alfa), has received marketing approval from the National Medical Products Administration (NMPA) as a long-term treatment for Pompe disease. This marks Sanofi’s second drug approved for Pompe disease in China, following the approval of Myozyme (alglucosidase alfa) in October 2015.
Understanding Pompe Disease and the Role of Nexviazyme
Pompe disease is caused by a deficiency in the enzyme acid α-glucosidase (GAA), leading to the intralysosomal accumulation of glycogen and subsequent muscular atrophy. Nexviazyme is a glycogen-specific recombinant GAA that features mannose-6-phosphate (M6P) moieties. These moieties enhance binding to surface receptors, facilitating increased internalization into cells, where they can more effectively restore GAA activity compared to Myozyme.
Nexviazyme was first approved in the United States in August 2021 for the treatment of late-onset Pompe disease in patients aged 1 year and older. This approval followed its designation as a breakthrough therapy by the FDA, along with fast-track and priority review statuses. In August 2022, Nexviazyme received priority review status in China, paving the way for its recent approval.-Fineline Info & Tech
