Press Release: Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease
View original at globenewswire.comPress 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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Additional analyses of the PERIDOT data are ongoing with more information to be shared at a future medical meeting
80% confidenceVenglustat performed as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume, and hemoglobin levels
80% confidenceA daily pill could make a serious difference for Gaucher patients facing neurological challenges
80% confidenceVenglustat was well tolerated overall with no new safety signals compared with previous studies
80% confidenceSanofi will pursue global regulatory filings for venglustat in GD3
80% confidenceCerezyme can now be prescribed globally to patients with either GD1 or GD3
80% confidenceThere are no approved treatments for neurologic manifestations of GD3
80% confidenceThese findings underscore Sanofi's commitment to rare disease research and the promise we aim to deliver for people living with these conditions
80% confidence
Data points we hold from this source
| Sanofi · rare disease commitment | 40 years |