Agamree

(Vamorolone)

ApprovedSmall molecule

CCatalyst Pharmaceuticals, Inc.

Agamree is one of 24 small molecule programs targeting Duchenne Muscular Dystrophy. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Duchenne Muscular DystrophyActive, not recruiting

Duchenne Muscular DystrophyApproved

Active, not recruiting

Competition

Competitive Landscape

40 competitors in Duchenne Muscular Dystrophy

#DrugCompanyPhaseTrials

Isordil

AstraZeneca

Approved

—2

Deltasone

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Approved

2 3

Coreg

Akero Therapeutics

Approved

1 4

Rapamune

PPrism Pharmaceuticals

Approved

1 5

Garamycin

Advil

Rifadin

1 8

Duvyzat

Italfarmaco

Phase 3

2 9

Vyondys 53

Sarepta Therapeutics

Approved

2 10

Agamree

CCatalyst Pharmaceuticals, Inc.

Approved

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2028

Post-ApprovalActive, not recruiting

Duchenne Muscular Dystrophy

NCT06713135

Q1 2032

Recruiting

Duchenne Muscular Dystrophy

NCT06564974

Clinical

Trials by Indication[1]

Duchenne Muscular Dystrophy5 trials

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Completed·SSanthera Pharmaceuticals·Phase 2·NCT05185622·54 enrolled

A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

Active, not recruiting·SSanthera Pharmaceuticals·Phase 4·NCT06713135·80 enrolled

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Completed·RReveraGen BioPharma, Inc.·Phase 2·NCT03439670·121 enrolled

Show 2 more trials

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Available·SSanthera Pharmaceuticals··NCT03863119

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]

Recruiting·CCatalyst Pharmaceuticals, Inc.··NCT06564974·250 enrolled

Midazolam1 trial

Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics

Completed·SSanthera Pharmaceuticals·Phase 1·NCT06689527·18 enrolled

Becker muscular dystrophy1 trial

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

Completed·RReveraGen BioPharma, Inc.·Phase 2·NCT05166109·46 enrolled

Unspecified1 trial

Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects

Completed·SSanthera Pharmaceuticals·Phase 1·NCT06649409·30 enrolled

Regulatory

FDA Approval Timeline[4]

Oct 26, 2023

AGAMREE

NDA NDA215239

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for Vamorolone. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Vamorolone compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Vamorolone. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA215239). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Agamree. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Catalyst Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: CCatalyst Pharmaceuticals, Inc.

Patent Landscape[5]

Patent Cliff

May 28, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10857161May 28, 2029

8334279May 28, 2029

11833159May 28, 2029

11690853Mar 7, 2033

12201639Mar 17, 2040

2 more patents

11471471Mar 17, 2040

11382922Jul 16, 2040

Agamree

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2028

Q1 2032

Trials by Indication[1]

Duchenne Muscular Dystrophy5 trials

Midazolam1 trial

Becker muscular dystrophy1 trial

Unspecified1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Related News