Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Genzyme, a Sanofi Company
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
38Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT02843035CT.gov Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Gaucher Disease Type I
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Venglustat (this asset) | Genzyme, a Sanofi Company | P2 | CCC · 53 |
| .Alpha.-D-Glucosyl-N-Acylsphingosine Glucohydrolase | Sanofi | P4 | A |
| Velaglucerase Alfa | Baylor Research Institute | P4 | BB |
| Miglustat | Actelion | P3 | BB |
| flt201 | Spur Therapeutics | P3 | B |
| Eliglustat | Genzyme, a Sanofi Company | P2 | B |
| Acetylcysteine | University of Minnesota | P2 | B |
| Ambroxol | Exsar Corporation | P1/2 | CCC |
| Ly-M001 | Lingyi Biotech Co., Ltd. | P1/2 | CCC |
+2 more in the Gaucher Disease Type I cohort
Other indications for Venglustat
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Fabry Disease | — | P3 | B · 61 |
| Tay-Sachs Disease | — | P3 | B · 58 |
| Sandhoff Disease | — | P3 | B · 58 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Venglustat in Gaucher Disease Type I. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/venglustat-gaucher-disease-type-i
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →