Tanruprubart Medicine 03 December 2024 Extended indication Guillain-Barré Syndrome Therapeutic value No estimate possible yet Total cost € 50,000,000.00 Registration phase Registration application pending Product Active substance Tanruprubart Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Muscular diseases other Extended indication Guillain-Barré Syndrome Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Annexon Portfolio holder Mechanism of action Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Complement C1 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date January 2026 Expected Registration February 2027 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options IVIg is de therapie van eerste keuze bij de behandeling van Guillain-Barré Syndrome (GBS). Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De uitkomsten van deze studie lieten een U-shaped effect zien met effect in de arm met lage dosis en minder goede (en met placebo vergelijkbare) effecten in de hoge dosis arm. De (lage) 30 mg/kg dosering lijkt dus optimaal; de oorzaak van het onverwachte effect in de hoge dosis groep is nog niet bekend. Aangezien het een eenmalige behandeling is en de prijs daarmee wellicht acceptabel kan blijven zal het potentieel IVIg kunnen gaan vervangen indien effectiviteit bevestigd wordt. Duration of treatment Frequency of administration Dosage per administration 30mg/kg of 75mg/kg References NCT04701164 (ANX005-GBS-02); Expertopinie Additional remarks Expected patient volume per year Patient volume < 200 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Richtlijnendatabase.nl Additional remarks GBS is de meest frequente oorzaak van acute spierzwakte in de westerse wereld. De incidentie in Nederland bedraagt 1,3 op de 100.000 per jaar. Dat betekent dat er in Nederland per jaar ongeveer 200 mensen GBS krijgen. Expected cost per patient per year Cost € 250,000.00 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 Additional remarks Kosten op basis van IHSI-inschatting, gebaseerd op de historische prijs van de ATC-klasse (L04AJ) van €250.000 per patiënt. Potential total cost per year Total cost € 50,000,000.00 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