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Source document· February 2, 2026

Press Release: Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease

View original at globenewswire.com
Press Release: Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease In the LEAP2MONO phase 3 study, venglustat, dosed orally once daily, demonstrated clinically meaningful efficacy in patient…
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  • There are no approved treatments for neurologic manifestations of GD3

    80% confidence

  • These findings underscore Sanofi's commitment to rare disease research and the promise we aim to deliver for people living with these conditions

    80% confidence

  • Sanofi will pursue global regulatory filings for venglustat in GD3

    80% confidence

  • A daily pill could make a serious difference for Gaucher patients facing neurological challenges

    80% confidence

  • Cerezyme can now be prescribed globally to patients with either GD1 or GD3

    80% confidence

  • Additional analyses of the PERIDOT data are ongoing with more information to be shared at a future medical meeting

    80% confidence

  • Venglustat performed as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume, and hemoglobin levels

    80% confidence

  • Venglustat was well tolerated overall with no new safety signals compared with previous studies

    80% confidence

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Sanofi · rare disease commitment40 years
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