Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
Idorsia Pharmaceuticals Ltd.
Modality
Small molecule
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
56Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03737214CT.gov A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease | — | Active Not Recruiting | — | — | — |
NCT03425539CT.gov Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease | — | Completed | — | — | — |
NCT02930655CT.gov A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Fabry Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Lucerastat (this asset) | Idorsia Pharmaceuticals Ltd. | P3 | BB · 67 |
| Recombinant Alpha-Galactosidase A | Shire | P4 | A |
| Agalsidase Beta | Sanofi | P4 | A |
| Migalastat | Amicus Therapeutics | P3 | BBB |
| Pegunigalsidase Alfa | Chiesi Italia | P3 | BBB |
| Montelukast | Sanofi | P4 | BB |
| Fexofenadine | Sanofi | P4 | BB |
| Loratadine | Sanofi | P4 | BB |
| Dexasone | Sanofi | P4 | BB |
+28 more in the Fabry Disease cohort
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. Lucerastat in Fabry Disease. 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/lucerastat-fabry-disease
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