Company: SNY
Filing Date: 2025-02-13
Form Type: 20-F
Source: 0001121404-25-000010
Chunk: 63

Company: Sanofi
Filing Date: 2025-02-13
Form: 20-F
Chunk 63
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adyme Nexviazyme / Nexviadyme (avalglucosidase alfa-ngpt) is a novel mannose-6-phosphate (M6P) enriched enzyme replacement therapy (ERT) treatment designed as a monotherapy for the entire spectrum of infantile-onset and late-onset Pompe disease (IOPD, LOPD), including patients who have changed treatments and naive patients, who have not received treatment previously. Nexviazyme/Nexviadyme is scientifically designed to specifically target the M6P receptor, the key pathway for ERT, to effectively clear glycogen build-up in muscle cells. It helps replace the GAA enzyme for people whose bodies do not produce enough. Investment in the clinical development of Nexviazyme is continuing, with an ongoing Phase 3 study in treatment-naive IOPD patients aged less than 12 months. Nexviazyme/Nexviadyme is administered as a monotherapy ERT every two weeks. Nexviazyme was first approved in the US by the FDA on August 6, 2021 for LOPD patients aged one year and older. On June 24, 2022, the EC granted marketing authorization for Nexviadyme as a potential new standard of care for the long-term treatment of both LOPD and IOPD. Nexviazyme/Nexviadyme has been approved in more than 59 countries and successfully launched in 32 countries including the US, Germany, the UK, other European markets, Japan and Australia. In all launched markets, the vast majority of eligible patients are currently being treated with Nexviazyme/Nexviadyme. Fabrazyme Fabrazyme (agalsidase beta) is an ERT used to treat Fabry disease (FD). FD is a multisystemic, progressive, X-linked inherited disorder of glycosphingolipid metabolism due to deficient or absent lysosomal α-galactosidase A activity resulting in progressive globotriaosylceramide (GL-3) accumulation in the lysosomes of various tissues. FD affects both genders . With age, progressive organ damage develops, leading to potentially life-threatening renal, cardiac and/or cerebrovascular complications. FD is characterized by different symptom severities and rates of progression, ranging from classic disease with early symptom onset to non-classic disease with cardiac and/or renal complications later in life. FD is seen in all racial and ethnic groups and is an under- diagnosed condition.