Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
Santhera Pharmaceuticals
Modality
Small molecule
Development Phase
Phase 4 / Approved
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
57High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06713135CT.gov A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy | — | Active Not Recruiting | — | — | — |
NCT06564974CT.gov Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT | — | Recruiting | — | — | — |
NCT05185622CT.gov A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) | — | Completed | — | — | — |
NCT03439670CT.gov A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | — | Completed | — | — | — |
NCT03038399CT.gov Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | — | Completed | — | — | — |
NCT02760277CT.gov An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | — | Completed | — | — | — |
NCT02760264CT.gov A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | — | Completed | — | — | — |
NCT03863119CT.gov Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy | — | Available | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Duchenne Muscular Dystrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Vamorolone (this asset) | Santhera Pharmaceuticals | Approved | BB · 67 |
| Viltolarsen | NS Pharma, Inc. | P4 | BBB |
| Golodirsen | Rare Disease Research, LLC | P4 | BB |
| Aoc 1044 | Avidity Biosciences, Inc. | P3 | BB |
| Delandistrogene Moxeparvovec | Sarepta Therapeutics, Inc. | P3 | BB |
| Prednisolone | Anne M. Connolly | P4 | BB |
| Eteplirsen | Sarepta Therapeutics, Inc. | P3 | BB |
| Glutamine | Assistance Publique - Hôpitaux de Paris | P3 | BB |
| Casimersen | Kevin Flanigan | P3 | BB |
+41 more in the Duchenne Muscular Dystrophy cohort
Other indications for Vamorolone
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Becker Muscular Dystrophy | — | P2 | B · 57 |
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. Vamorolone in Duchenne Muscular Dystrophy. 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/vamorolone-duchenne-muscular-dystrophy
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