(RTTNews) - Sanofi (SNY, SAN.PA, SNYNF) announced that the US Food and Drug Administration has granted Breakthrough Therapy designation to Venglustat for the treatment of neurological manifestations of type 3 Gaucher disease based on LEAP2MONO phase 3 study data.
Gaucher disease (GD) is a rare inherited lysosomal storage disorder that results from a deficiency of the enzyme called glucocerebrosidase, leading to the accumulation of sugar- and fat-molecules called glycosphingolipids (GSL) in the spleen, liver, bone marrow, and lungs. There are three types: GD1, GD2, and GD3.
GD1 is characterised by a lack of central nervous system involvement; GD2, by rapid neurological decline and severe neurocognitive symptoms; and GD3, by slower, more variable progression and symptom severity.
Venglustat is a novel, investigational oral glucosylceramide synthase inhibitor (GCSi) designed to cross the blood-brain barrier and has the potential to slow the progression of GD3 by inhibiting abnormal GSL accumulation and its physico-pathologic consequences.
The LEAP2MONO phase 3 study was a double-blind, double-dummy, active-comparator, two-arm study that evaluated the efficacy and safety of once-daily oral Venglustat versus intravenous ERT every 2 weeks in adults and paediatric patients aged 12 and older with GD3.
43 patients were randomised in a 1:1 ratio to receive Venglustat and placebo infusion or ERT and placebo tablet.
In the LEAP2MONO phase 3 study, patients receiving Venglustat demonstrated statistically significant improvements in neurological symptoms, as measured by a global test score that included assessments of ataxia (mSARA) and cognition (RBANS), compared with patients receiving the enzyme replacement therapy (ERT), Imiglucerase, at week 52.
In addition, Venglustat was well tolerated overall, with no new safety signals observed compared with previous studies.
The LEAP2MONO study is ongoing, and results from its open-label phase will be shared in future.
Sanofi will continue global regulatory filings for Venglustat in GD3 during 2026. Venglustat previously received fast-track designation from the FDA for its potential use in GD3, as well as orphan designation for GD3 in the US, EU, and Japan.
SNY has traded between $43.34 and $59.13 in the last year. The stock closed Tuesday's trade at $44.06, up 0.8%.
In the premarket trade, SNY is up 0.49% at $43.84.
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