Deferiprone
Friedreich Ataxia
Floatz Rating
BB66/100
Confidence
Indicative
Sponsor
ApoPharma
Modality
Small molecule
Development Phase
Phase 2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
51High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT00897221CT.gov A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia | — | Completed | — | — | — |
NCT00530127CT.gov A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia | — | Completed | — | — | — |
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 · Friedreich Ataxia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Deferiprone (this asset) | ApoPharma | P2 | BB · 66 |
| Idebenone | Santhera Pharmaceuticals | P3 | A |
| Interferon Γ-1B | Amgen | P3 | BB |
| Omaveloxolone | Biogen | P3 | BB |
| rt001 | Biojiva LLC | P3 | BB |
| Trans-Resveratrol | Murdoch Childrens Research Institute | P2 | BB |
| Citalopram | University of Colorado, Denver | P4 | BB |
| Vatiquinone | PTC Therapeutics | P3 | BB |
| L-Methionyl-L-Tyrosylglycyl-L-Arginyl-L-Lysyl-L-Lysyl-L-Arginyl-L-Arginyl-L-Glutaminyl-L-Arginyl-L-Arginyl-L-Arginylglycylglycine Fusion Protein With Frataxin (Human Isoform 1 Precursor) | Larimar Therapeutics, Inc. | P2 | BB |
+30 more in the Friedreich Ataxia cohort
Other indications for Deferiprone
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta-Thalassemia Major | — | P4 | BBB · 77 |
| Iron Metabolism Disease | — | P4 | BBB · 73 |
| Hemosiderosis | — | P4 | BB · 71 |
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.
Citation
Floatz Terminal. Deferiprone in Friedreich Ataxia. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 13, 2026. https://terminal.floatz.ai/assets/deferiprone-friedreich-ataxia
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