Sanofi Says Venglustat Meets Primary Endpoints in Phase 3 Study of Type 3 Gaucher Disease

MT Newswires Live
02/02

Sanofi (SNY) said Monday venglustat met its primary goal in a phase 3 study involving adult and pediatric patients with neurological symptoms of type 3 Gaucher disease, but failed to meet the main goal in another study.

The company has been evaluating venglustat for type 3 Gaucher disease and Fabry disease, both rare inherited lysosomal storage diseases.

Sanofi said patients with type 3 Gaucher disease treated with venglustat showed statistically significant improvements in neurological function at 52 weeks, based on global assessment scores for ataxia and cognitive performance, compared with those receiving standard enzyme-replacement therapy.

The drug also performed as well as enzyme-replacement therapy on non-neurological outcomes, such as spleen and liver size and hemoglobin levels, meeting three of four key secondary endpoints, it added.

The drugmaker also said a phase 3 study of the drug in patients with Fabry disease showed reductions in neuropathic and abdominal pain, but the trial did not meet its primary endpoint.

The company said it plans to pursue global regulatory filings for venglustat in type 3 Gaucher disease.

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