Company: SNY
Filing Date: 2025-02-13
Form Type: 20-F
Source: 0001121404-25-000010
Chunk: 87

Company: Sanofi
Filing Date: 2025-02-13
Form: 20-F
Chunk 87
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 drug designation in the US and in Europe. In 2024, regulatory submissions for the treatment of hemophilia A or B in adults and adolescents with or without inhibitors were completed in several regions, including the US with a prescription drug user fee act (PDUFA) date of March 28, 2025. In addition, Sanofi’s collaboration partner Siemens Healthineers submitted the INNOVANCE ® Antithrombin Assay for FDA review as a companion diagnostic that will measure antithrombin levels in people living with hemophilia who are prescribed fitusiran. rilzabrutinib (SAR444671) is a Bruton’s tyrosine kinase inhibitor (see details in section “— a) Immunology & Inflammation” above) developed for the treatment of immune thrombocytopenia (ITP), for which the FDA has granted fast-track designation. The asset has also obtained orphan drug designation in the US, Europe, and Japan. The primary endpoint of durable platelet response was met in the rilzabrutinib Phase 3 study LUNA 3 in adult patients with persistent or chronic ITP. Other key secondary endpoints were met including reduced bleeding, number of weeks with platelet response, the need for rescue therapy use, and improved physical fatigue and quality of life measures. The safety profile of rilzabrutinib was favorable and consistent with that reported in previous studies. Rilzabrutinib is under regulatory review in the EU, China, and the US with a target date to receive an FDA decision on August 29, 2025. In 2024, a Phase 2 study in warm autoimmune hemolytic anemia read out positively with clinically meaningful outcomes on response rate and additional disease markers. The results of this study build on the successful Phase 3 study of rilzabrutinib in ITP and reinforce its efficacy in autoimmune cytopenias . venglustat (GZ402671) is an orally administered brain penetrant glucosylceramide synthase (GCS) inhibitor that blocks the conversion of ceramide to glucosylceramide (GL-1). In 2024, the AMETHIST Phase 3 study of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints. The data reinforced the favorable safety profile and do not impact the other two indications, FD and Gaucher disease type