Vamorolone

Orphan DrugFDA Approved

Description

Vamorolone is an oral dissociative corticosteroid approved for Duchenne muscular dystrophy. It was developed to preserve anti-inflammatory activity while potentially reducing some steroid-related toxicities, making it a differentiated specialty option in an orphan neuromuscular disease. Global access has expanded through multiple regulatory pathways outside the United States as well.

Indications & Therapeutic Use

Duchenne muscular dystrophy

Linked Diseases:

Global Availability (2 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Vamorolone
Generic Namevamorolone
Brands1 brand available
Active Ingredientvamorolone
Drug ClassDuchenne muscular dystrophy
ManufacturerSanthera Pharmaceuticals
Dosage FormsOral suspension
Medical CodeH02AB
Orphan StatusYes — Orphan Drug
Cold ChainNot Required
Reg. StatusFDA Approved
Clinical TrialNCT02760264
Countries2 countries
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations3 Validated Nodes