What were the results of Sanofi's venglustat trial for type 3 Gaucher disease?

Venglustat showed superior improvements in neurological symptoms like speech and limb coordination for type 3 Gaucher disease patients compared to enzyme replacement therapy.

Did Sanofi's venglustat show success in the Fabry disease trial?

Venglustat helped reduce neuropathic and abdominal pain in Fabry disease patients, but did not meet the study's main goal for statistical success.

What is venglustat designed to treat?

Venglustat is an experimental oral drug being tested for rare genetic disorders like type 3 Gaucher disease and Fabry disease, which are caused by enzyme deficiencies.

Home / Health / Sanofi Drug Shows Mixed Results in Genetic Disease Trials

Sanofi Drug Shows Mixed Results in Genetic Disease Trials

2 Feb

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Summary

Experimental drug venglustat showed promise in type 3 Gaucher disease.
The treatment did not meet its main goal in a Fabry disease trial.
Sanofi's oral treatment targets rare inherited enzyme deficiency disorders.

Sanofi Drug Shows Mixed Results in Genetic Disease Trials

Sanofi announced mixed outcomes for its experimental genetic disorder treatment, venglustat. The drug showed promise in a late-stage study involving patients with type 3 Gaucher disease, significantly improving neurological symptoms like speech and coordination compared to existing therapies.

However, venglustat did not achieve statistical success in a concurrent trial for Fabry disease. While it aided in reducing neuropathic and abdominal pain, the results were not conclusive, potentially due to a strong placebo effect. Sanofi is analyzing the data and consulting with global regulators.

This experimental oral therapy is designed to combat inherited conditions stemming from enzyme deficiencies that cause toxic substance buildup. Sanofi is banking on its late-stage pipeline, including venglustat, to drive future sales growth, especially as its leading eczema and asthma drug approaches patent expiration.