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Source document· March 18, 2026

Communiqué de presse : Le venglustat de Sanofi a reçu la désignation de thérapie innovante aux États-Unis pour la maladie de Gaucher de type 3

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Communiqué de presse : Le venglustat de Sanofi a reçu la désignation de thérapie innovante aux États-Unis pour la maladie de Gaucher de type 3 Le venglustat de Sanofi a reçu la désignation de thérapie innovante aux États-Unis pour la maladie de Gaucher de type 3 Paris, le 18 mars 2026…

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There is currently no approved treatment for the neurological symptoms of Gaucher disease type 3
60% confidence
Venglustat was generally well tolerated with no new safety signals compared to previous studies
60% confidence
There is no approved treatment for neurological symptoms of Gaucher disease type 3
60% confidence
The positive results from the LEAP2MONO study are an encouraging advancement in the research and development process
60% confidence
Venglustat works by reducing abnormal accumulation of glycosphingolipids and is designed to cross the blood-brain barrier to target the underlying pathology causing neurological effects of GD3
60% confidence
This regulatory milestone recognizes the significant unmet medical need for people living with Gaucher disease type 3, particularly those experiencing progressive neurological deterioration
60% confidence
Venglustat was generally well tolerated with no new safety signals compared to previous studies
60% confidence
Sanofi will continue to collaborate with the FDA to advance this potential therapeutic option
60% confidence
The positive results from the LEAP2MONO study are an encouraging advance in the research and development process, and we will continue to collaborate with the FDA to advance this potential treatment option
60% confidence
This regulatory milestone recognizes the significant unmet medical need for people living with Gaucher disease type 3, particularly those experiencing progressive neurological deterioration
60% confidence
Venglustat has the potential to slow the progression of Gaucher disease type 3 by inhibiting abnormal accumulation of glycosphingolipids and its pathophysiological consequences
60% confidence