Extended indication Treatment of Duchenne muscular dystrophy.
Therapeutic value Possible added value
Registration phase Clinical trials

Product

Active substance Vamorolone
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Duchenne
Extended indication Treatment of Duchenne muscular dystrophy.
Manufacturer ReveraGen
Route of administration Oral
Therapeutical formulation Oral suspension
Budgetting framework Extramural (GVS)
Additional comments Glucocorticoid receptor agonist; Mineralocorticoid receptor antagonist.

Registration

Registration route Centralised (EMA)
Submission date 2022
Expected Registration 2023
Orphan drug Yes
Registration phase Clinical trials
Additional comments Primary completion datum fase 2 studie in februari 2022.

Therapeutic value

Current treatment options Corticosteroïden.
Therapeutic value Possible added value
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.
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 comments 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.

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 comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References Adisinsght
Additional comments Enkel preklinische studies.

Other information

There is currently no futher information available.