The FDA granted Breakthrough Therapy designation to Sanofi’s investigational oral glucosylceramide synthase inhibitor venglustat for neurological manifestations of type 3 Gaucher disease. The designation was based on results from the LEAP2MONO phase 3 study (NCT05222906), in which venglustat showed statistically significant improvement on a global neurological test score versus imiglucerase (p=0.007). Sanofi said venglustat was generally well tolerated in the study, with headache reported in 14.3% of patients on venglustat and 18.2% on imiglucerase. The company said it plans to pursue global regulatory filings for venglustat in type 3 Gaucher disease during 2026.

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