Company: SNY
Filing Date: 2025-02-13
Form Type: 20-F
Source: 0001121404-25-000010
Chunk: 62

Company: Sanofi
Filing Date: 2025-02-13
Form: 20-F
Chunk 62
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 in the EU since 1997, in Japan since 1998 and in China since 2008, and is approved to treat type 1 Gaucher disease in more than 85 countries. It has also been approved to treat the systemic symptoms of type 3 Gaucher disease in most non-US markets, including the EU and Japan. Cerdelga Cerdelga (eliglustat) is the first and only first-line oral therapy for Gaucher disease type 1 adult patients. A potent, highly specific ceramide analog inhibitor of GL-1 synthesis with broad tissue distribution, Cerdelga has demonstrated efficacy in the treatment of naive Gaucher disease patients and in patients who switch from enzyme replacement therapy. Cerdelga has been approved to treat type 1 Gaucher disease in the US (2014), and in the EU and Japan (2015). It is also in development for the treatment of type 1 Gaucher disease in pediatric patients. See “— B.4. Global Research & Development.” Myozyme and Lumizyme Myozyme (alglucosidase alfa) is an ERT used to treat both Infantile Onset and Late Onset Pompe disease (IOPD and LOPD). Pompe disease is an inherited, progressive and often fatal neuromuscular disease, caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) that results in the build-up of glycogen in the muscles’ cells. For IOPD, symptoms begin within a few months of birth and there are impacts on the heart in addition to causing skeletal muscle weakness. Other symptoms include difficulties breathing, frequent chest infections, problems feeding that result in failure to gain weight as expected, and failure to meet certain developmental milestones. Patients with LOPD typically present symptoms any time after the first year of life to late adulthood and rarely manifest cardiac problems. The hallmark symptom of LOPD is skeletal muscle weakness, which often leads to walking disability and reduced respiratory function. Patients often require wheelchairs to assist with mobility and may require mechanical ventilation to help with breathing. Pompe disease occurs in approximately one in 40,000 newborns worldwide, but incidence and patient severity vary among regions. Myozyme was first approved in 2006 in the EU and has since been approved in more than 80 countries. In the US, alglucosidase alfa has been marketed as Lumizyme since 2010. Nexviazyme/Nexvi