Vamorolone

Treats Duchenne muscular dystrophy (DMD). Sold as Agamree. Every FDA application for this active ingredient.

As of Nov 15, 2026, FDA lists 1 application for Vamorolone (sold as Agamree), first approved Oct 26, 2023, with 0 new uses approved and 0 published complete response letters.

First approved: Oct 26, 2023
Applications: 1
New uses approved: 0
Complete response letters: 0
Medicare spending, 2025: $2.1M
Orphan exclusivity until: Oct 26, 2030
First approved in the EU: Dec 14, 2023

Medicare Spending

CMS Part D and Part B, 2025, before manufacturer rebates; matched by generic name

Product	Part	Spending, 2025	Change	Beneficiaries
Agamree	Part D	$2.1M	+202%	19

Who Was in the Trials

118 participants in the largest population FDA reports for the Oct 26, 2023 approval, from FDA's Drug Trials Snapshot; see every snapshot

Women: 0%
65 and older: Not reported
White: 83%
Black: 2%
Asian: 10%
Hispanic or Latino: 4%

Orphan Designations (FDA)

2 designations, 1 approved for the designated use; see all orphan designations

Treatment of Becker muscular dystrophyReveraGen BioPharma Inc. · designated Dec 18, 2023
Treatment of Duchenne muscular dystrophyCatalyst Pharmaceuticals, Inc. · designated Dec 2, 2011 · approved Oct 26, 2023 · exclusive to Oct 26, 2030

Approvals in the EU (EMA)

First approved in the EU 2 months after the US. Centrally authorised medicines, matched by ingredient; see all EU approvals

Agamree, new substanceSanthera Pharmaceuticals (Deutschland) GmbH · Dec 14, 2023

Clinical Trials

2 open or ongoing studies name this ingredient as an intervention, including as a comparator; newest first. See all


A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy · Santhera Pharmaceuticals	Phase 4	Active, not recruiting	Dec 3, 2024
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMITDuchenne Muscular Dystrophy · Catalyst Pharmaceuticals, Inc.	—	Recruiting	Aug 21, 2024

Applications

NDAs, BLAs and generic ANDAs for this ingredient, oldest first; each opens its full history

Approved	Application	Sponsor	Supplements	Marketing
Oct 26, 2023	AgamreeNDA 215239 · New molecular entity	Catalyst Pharms	1	Marketed

Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA