Ponesimod Medicine 10 December 2019 Extended indication Treatment of Relapsing-remitting multiple sclerosis (MS) Therapeutic value Possible equal value Total cost € 4,900,000.00 Registration phase Registration application pending Product Active substance Ponesimod Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Multiple sclerosis Extended indication Treatment of Relapsing-remitting multiple sclerosis (MS) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Janssen Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Coated tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Oral selective sphingosine-1 phosphate-1 receptor agonist Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2020 Expected Registration May 2021 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options teriflunomide (vergelijkende behandeling studie) en naar verwachting de andere orale MS-middelen zoals fingolimod (zie farmacotherapeutisch kompas). Therapeutic value Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation Tussen de sfingosine-1-fosfaat receptoragonisten is veel concurrentie en weinig verschil in aangetoonde effectiviteit. Het verschil tussen deze geneesmiddelen ligt met name in de bijwerkingsprofielen. Duration of treatment Frequency of administration 1 times a day Dosage per administration 20 mg References OPTIMUM (NCT02425644) Additional remarks Expected patient volume per year Patient volume 200 - 500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIP databank, hersenstichting, Nationaal MS fonds. Additional remarks Hersenstichting: In Nederland komt MS voor bij ongeveer 1 op de 1.000 inwoners. Nederland telt dus ongeveer 17.000 mensen met MS. RMS komt bij ongeveer 40% van alle mensen met MS voor (6.800 personen). Door de concurrentie aan middelen zal het verwachte marktaandeel op maximaal een paar honderd patiënten komen te liggen. Expected cost per patient per year Cost € 9,000.00 - 19,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GIP-databank Additional remarks Er is nog geen prijs bekend maar de verwachting is dat dit in lijn zal zijn met directe concurrenten. Bij substitutie geen toename in kosten. Potential total cost per year Total cost € 4,900,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References adis insight, clinicaltrials.gov Additional remarks Graft-versus-host disease (fase II) Other information Additional remarks