Product Description
Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. This has the potential to 'dissociate' efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from patient quality of life. On September 2, 2020, Santhera exercised its option and obtained worldwide rights to vamorolone in Duchenne muscular dystrophy and all other indications. (Sourced from: https://www.santhera.com/health-care-professionals/vamorolone)
Mechanisms of Action: Steroid receptor Agonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Priority Review - Muscular Dystrophies|Muscular Dystrophy, Duchenne *
Approval Status: Approved
Approved Countries: Czech
Approved Indications: None
Known Adverse Events: None
Company: ReveraGen BioPharma
Company Location:
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Canada, China, Germany, Italy, United States
Active Clinical Trial Count: 7
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Catalyst Biosciences presented P2 Muscular Dystrophy, Duchenne results on 2024-03-03 for Vamorolone
- Clinical Outcomes Reported - Catalyst Biosciences presented P2 Muscular Dystrophy, Duchenne results on 2024-02-21 for Vamorolone
- Vamorolone's PDUFA date is set for October 26, 2023, for Duchenne muscular dystrophy treatment approval by the FDA.
Highest Development Phases
Phase 2: Muscular Dystrophy, Becker|Muscular Dystrophy, Duchenne
Phase 1: Drug Hypersensitivity|Healthy Volunteers
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05166109 |
VBP15-BMD-001 | P2 |
Completed |
Muscular Dystrophy, Becker|Muscular Dystrophy, Duchenne |
2025-08-18 |
50% |
2025-09-19 |
|
NCT06689527 |
SNT-I-VAM-025 | P1 |
Completed |
Drug Hypersensitivity |
2024-09-24 |
2024-11-15 |
Primary Endpoints|Treatments |
|
CTR20232865 |
CTR20232865 | P1 |
Completed |
Muscular Dystrophy, Duchenne |
2024-01-08 |
2025-04-29 |
||
2025-000201-16 |
006 | P2 |
Completed |
Muscular Dystrophy, Duchenne |
2024-05-07 |
2025-07-09 |
Primary Completion Date|Start Date|Study Completion Date|Treatments |
|
2022-000844-31 |
Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy | P2 |
Completed |
Muscular Dystrophy, Becker |
2025-05-18 |
2025-05-06 |
||
NCT05185622 |
VBP15-006 | P2 |
Completed |
Muscular Dystrophy, Duchenne |
2024-07-16 |
50% |
2025-10-28 |
|
NCT06649409 |
SNT-I-VAM-026 | P1 |
Completed |
Healthy Volunteers |
2024-06-29 |
2024-10-19 |
Primary Endpoints|Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
11/05/2025 |
News Article |
Santhera Closes USD 13 Million Royalty Monetization Agreement |
|
11/04/2025 |
News Article |
Five-year data of AGAMREE® (vamorolone) in patients with DMD show improved safety profile with comparable effectiveness to standard of care corticosteroids |
|
10/09/2025 |
News Article |
Santhera Secures Agreement with Biomedica for the Distribution of AGAMREE® (Vamorolone) in Russia |
|
10/03/2025 |
News Article |
Santhera Announces Approval in Canada for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy |