Natalizumab Medicine 08 December 2020 Extended indication "Tysabri is indicated as single disease modifying therapy in adults with highly active relapsing remitting multiple sclerosis for the following patient groups: •Patients with highly active disease activity despite a full and adequate course of treatment with at least one disease modifying therapy (DMT) (for exceptions and information about washout periods) •Patients with rapidly evolving severe relapsing remitting multiple sclerosis defined by 2 or more disabling relapses in one year, and with 1 or more Gadolinium enhancing lesions on brain MRI or a significant increase in T2 lesion load as compared to a previous recent MRI. Therapeutic value Possible equal value Total cost € 8,100,000.00 Registration phase Clinical trials Product Active substance Natalizumab Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Multiple sclerosis Extended indication "Tysabri is indicated as single disease modifying therapy in adults with highly active relapsing remitting multiple sclerosis for the following patient groups: •Patients with highly active disease activity despite a full and adequate course of treatment with at least one disease modifying therapy (DMT) (for exceptions and information about washout periods) •Patients with rapidly evolving severe relapsing remitting multiple sclerosis defined by 2 or more disabling relapses in one year, and with 1 or more Gadolinium enhancing lesions on brain MRI or a significant increase in T2 lesion load as compared to a previous recent MRI. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Biogen Portfolio holder Mechanism of action Immunosuppression Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Natalizumab is een gehumaniseerd monoklonaal antilichaam gericht tegen het celadhesiemolecuul α4-integrine. Binding aan α4-integrine verhindert dat witte bloedcellen vanuit het bloed het centraal zenuwstelsel bereiken en dat de zenuwcellen en hun uitlopers door ontstekingen worden vernietigd. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Alle huidige ziekte-modificerende behandelingen (DMT's, disease modifying therapies) voor de onderhoudsbehandeling van patiënten met 'relapsing' multipele sclerose (RMS). Therapeutic value Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat de effectiviteit van de subcutane variant van natalizumab vergelijkbaar zal zijn met ofatumumab. Voor natalizumab zullen enkel JCV negatieve patiënten in aanmerking komen. Duration of treatment Frequency of administration 1 times every 6 weeks Dosage per administration 300 mg References NCT03689972 Additional remarks Expected patient volume per year Patient volume 300 - 600 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank Additional remarks In 2019 waren er 1.077 gebruikers van de intraveneuze variant van natalizumab. De verwachting is dat er een afname zal plaatsvinden doordat patiënten in de toekomst zullen overstappen op ocrelizumab en ofatumumab. Er komen in Nederland vermoedelijk 300 tot 600 patiënten in aanmerking voor deze behandeling. Expected cost per patient per year Cost € 18,000.00 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 GIPdatabank; https://www.ema.europa.eu/en/medicines/human/EPAR/tysabri#product-information-section Additional remarks In 2019 werd er per gebruiker €17.916 vergoed voor natalizumab IV. De verwachting is dat natalizumab SC in dezelfde prijsrange zal worden ingezet. Potential total cost per year Total cost € 8,100,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Adis Insight Additional remarks Momenteel geen lopende fase 3 studies naar andere indicaties. Other information Additional remarks