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Drug

Vamorolone

Approved · EMA
Early-to-mid development

Also known as Agamree.

RxNorm2669799ChEMBLCHEMBL2348780UNII8XP29XMB43
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label, Single-arm Study to Evaluate the CYP3A4 Induction Potential of Vamorolone on the Pharmacokinetics of Midazolam (a Sensitive CYP3A4 Probe) in Healthy Subjects.

Clinical trial2025-12-15Results posted · ClinicalTrials.gov

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Clinical trial2025-10-24Results posted · ClinicalTrials.gov

An Open-label, Randomized, 3-arm, Parallel-group, Positive- and Negative-arm Controlled Study to Evaluate the Mineralocorticoid Receptor Antagonism Effect of Vamorolone in Healthy Subjects

Clinical trial2025-09-11Results posted · ClinicalTrials.gov

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

Clinical trial2025-08-18Completed · ClinicalTrials.gov

Approval: Agamree (EMA)

Regulatory2023-12-14EMA

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

Clinical trial2023-03-09Results posted · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Vamorolone
Aliases & brands
Agamree
RxNorm CUI
2669799
ChEMBL ID
CHEMBL2348780
UNII
8XP29XMB43
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2023-12-14
Latest approval
2023-12-14
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2023-12-14Approval
Approval: Agamree (EMA)

Indication: Agamree is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.&nbsp;

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

5 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
5
registered trials across all phases
LATEST COMPLETION 2025
5
Completed
PHASE DISTRIBUTIONn = 5
Phase 12Phase 23

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

CompletedPH IIReveraGen BioPharma, Inc.n=462025

An Open-label, Single-arm Study to Evaluate the CYP3A4 Induction Potential of Vamorolone on the Pharmacokinetics of Midazolam (a Sensitive CYP3A4 Probe) in Healthy Subjects.

CompletedPH ISanthera Pharmaceuticalsn=182024

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

CompletedPH IISanthera Pharmaceuticalsn=542024

An Open-label, Randomized, 3-arm, Parallel-group, Positive- and Negative-arm Controlled Study to Evaluate the Mineralocorticoid Receptor Antagonism Effect of Vamorolone in Healthy Subjects

CompletedPH ISanthera Pharmaceuticalsn=302024

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

CompletedPH IIReveraGen BioPharma, Inc.n=1212021
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.