Vamorolone Medicine 07 June 2022 Extended indication Treatment of Duchenne muscular dystrophy. Therapeutic value Possible added 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 Treatment of Duchenne muscular dystrophy. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer ReveraGen Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Oral suspension Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Glucocorticoïd receptor agonist; Mineralocorticoïd receptor antagonist. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date October 2022 Expected Registration November 2023 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Primary completion datum fase 2 studie in februari 2022. Therapeutic value Current treatment options Corticosteroïden. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Resultaten fase 2 studie laten gelijke effectiviteit zien ten opzichte van corticosteroïden met mogelijke voordelen in groeiontwikkeling voor kinderen van 4 tot 7 jaar. Duration of treatment Frequency of administration 1 times a day Dosage per administration 2.0 - 6.0 mg/kg/day. References Mah JK, Clemens PR, Guglieri M, et al. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178 Additional remarks Oral administration of 2.0 - 6.0 mg/kg/day. Expected patient volume per year Patient volume < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Spierziekten Nederland; Clemens et al. JAMA Neurol. 2020;77(8):982-991 (1). Mah JK et al., (2022); JAMA Netw Open. 2022;5:e2144178 (2), Expert opinion (3) Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie (1). De groep waarop getest wordt in de klinische studies (4 tot 7 jaar) betreft slechts een deel van deze populatie (2). Naar schatting zal het dus gaan om 100 patiënten (3) 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Adisinsght Additional remarks Enkel preklinische studies. Other information Additional remarks