---
title: "Venglustat: Parkinson's Drug Pipeline"
description: "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."
canonical_url: "https://parkinsonspathways.com/pipeline/venglustat"
development_stage: Recently stopped or failed
last_updated: 2026-06-01
source: "Parkinson's Pathways"
---
# Venglustat

**Stage:** Phase 2 failed (2021)  ·  **Type:** Disease-modifying

**Developer:** Sanofi

**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.

**Target:** Glucosylceramide synthase / GBA pathway

**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

- [MOVES-PD on ClinicalTrials.gov](https://clinicaltrials.gov/study/NCT02906020)

_Last reviewed: 2026-06-01._

## More

- [Back to the Parkinson's drug pipeline](https://parkinsonspathways.com/pipeline)
- [Browse all recruiting trials](https://parkinsonspathways.com/trials)