Vamorolone Medicine 04 December 2025 Extended indication Extension of indication to include treatment of 2 to <4 year olds with Duchenne muscular dystrophy (DMD) Therapeutic value Total cost Registration phase Registration application pending Product Active substance Vamorolone Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Duchenne Extended indication Extension of indication to include treatment of 2 to <4 year olds with Duchenne muscular dystrophy (DMD) Current proprietary name Already available biosimilars / generics Proprietary name Agamree Manufacturer Santhera Portfolio holder Santhera Mechanism of action Route of administration Oral Therapeutical formulation Oral suspension Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date September 2025 Expected Registration July 2026 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks