Tolebrutinib Medicine 03 December 2024 Extended indication Relapsing forms of MS (RMS) and non-relapsing secondary progressive MS (nrSPMS) Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Tolebrutinib Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Multiple sclerosis Extended indication Relapsing forms of MS (RMS) and non-relapsing secondary progressive MS (nrSPMS) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sanofi Portfolio holder Sanofi Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Tolebrutinib is an investigational brain-penetrant and bioactive Bruton’s tyrosine kinase (BTK) inhibitor that achieves CSF concentrations predicted to modulate B lymphocytes and microglial cells. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date 2025 Expected Registration 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Ongoing phase 3 clinical trials: NCT04964791 + NCT04410991; NCT04411641; Registration expected in the first half of 2026. Therapeutic value Current treatment options Alemtuzumab, cladribine, fingolimod, glatiramer,interferon bèta-1a, interferon bèta-1b, natalizumab, ocrelizumab, peginterferon bèta-1a, teriflunomide Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er is een groep van 5 btk-remmers in ontwikkeling waarvan er twee in fase 3 zijn en vergeleken met teriflinumide komt geen voordeel uit voor tolebrutinib uit. Voor patiënten met secundair progressieve MS zonder relapses wordt er wel een duidelijk verschil gezien in voordeel van btk-remmers. Voor deze patiënten is er nu geen behandeling. Er is waarschijnlijk geen meerwaarde voor de MS relapsing remitting groep. Duration of treatment Frequency of administration Dosage per administration References https://www.sanofi.com/en/media-room/press-releases/2020/2020-04-23-05-00-00-2020541 https://www.sanofi.com/en/media-room/press-releases/2021/2021-10-13-07-00-00-2313117 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1: Multiple sclerose (MS). Hersenstichting. 2024. 2: Expertopinie Additional remarks 36.200 patiënten waren er in 2021 bekend met multiple sclerose (1). De verwachting is dat het medicament een rol kan gaan spelen bij niet actieve secundair progressieve MS met EDSS 3 tot 6,5 (waarvoor geen siponimod). Vooralsnog is de inschatting dat er maximaal 2.000 patiënten in aanmerking zullen komen (2). 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 No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions PPMS References Currently in phase 3 clinical trial: NCT04458051 Additional remarks Other information Additional remarks