Venglustat
Recently stopped or failed
A drug aimed at GBA-related Parkinson's that failed its Phase 2 trial in this group and was not advanced for Parkinson's. Included for an honest picture.
- Stage: Phase 2 failed (2021)
- Type: Disease-modifying
- Developer: Sanofi
- Target: Glucosylceramide synthase / GBA pathway
- How it works: A pill that reduces the build-up of certain fatty molecules by partially blocking the enzyme that makes them, aimed mainly at people with a GBA gene variant.
- Who it's for: People with Parkinson's who carry a GBA gene variant (GBA-PD).
- Why it stopped: The Phase 2 MOVES-PD trial in GBA-Parkinson's did not show benefit, and the Parkinson's program was not advanced.
- Key trials: NCT02906020
- Last reviewed: 2026-06-01
What it is
Venglustat targets the same GBA/lysosome pathway that ambroxol does, but from the opposite direction, reducing a fatty substrate rather than boosting the GCase enzyme. It was tested specifically in people whose Parkinson's is linked to a GBA gene variant.
Where it stands
The Phase 2 MOVES-PD trial in GBA-Parkinson's did not show benefit, and Sanofi did not move venglustat forward for Parkinson's. (It has been studied in other, unrelated conditions.)
What the data shows so far
MOVES-PD enrolled people with GBA-Parkinson's and, over the trial period, did not show a meaningful slowing of progression versus placebo on its main measures: a negative result in exactly the genetic subgroup it was designed for.
What families should know
Its failure is a reminder that targeting a known genetic pathway, even in the 'right' patients, does not guarantee benefit. It also shows why GBA-focused approaches (like ambroxol) still have to prove themselves in trials.
Caveats
Failed its Phase 2 trial in GBA-Parkinson's; not in development for Parkinson's. Listed only for transparency. Do not confuse it with ambroxol, which targets the same pathway differently and is still being tested.
Timeline
Most recent first.
- 2021, MOVES-PD reported as not showing benefit; not advanced for Parkinson's.
- 2017, MOVES-PD Phase 2 in GBA-Parkinson's begins.
Sources
Last reviewed: 2026-06-01. Back to the Parkinson's drug pipeline