Teplizumab Medicine 08 December 2020 Extended indication To prevent or delay the onset of type 1 diabetes (T1D) in relatives determined to be at very high risk for developing T1D aged 8 years and older. Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Teplizumab Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Diabetes Extended indication To prevent or delay the onset of type 1 diabetes (T1D) in relatives determined to be at very high risk for developing T1D aged 8 years and older. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Provention bio Portfolio holder Mechanism of action Other Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks CD3 antigen inhibitor Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat immuuntherapie niet effectief zal zijn bij het behandelen van diabetes type 1. Indien patiënten preventief behandeld zullen worden, betekent dit dat gezonde jonge patiënten met een geneesmiddel behandeld worden dat mogelijk bijwerkingen kan hebben. Duration of treatment Average 2 week / weeks Frequency of administration 1 times a day Dosage per administration References NCT01030861 Additional remarks Intravenous infusions of teplizumab given for 14 consecutive days. Each infusion takes about 30 minutes and is followed by a 2 hour observation period. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank; Additional remarks Totaal aantal gebruikers van diabetesmiddelen in 2016 was 814.714. 10% hiervan heeft diabetes type 1 dus het aantal patiënten in Nederland bedraagt ongeveer 80.000. De incidentie zal naar waarschijnlijkheid maximaal 2.000 patiënten bedragen. Eerstegraads familieleden tussen de 8 jaar-45 jaar en 2e graads familieleden tussen de 8 jaar-20 jaar komen in aanmerking, wanneer bevestigd is dat zij ten minste twee diabetes autoantibodies hebben. Er wordt voorlopig niet verwacht dat deze behandeling ingezet zal worden in Nederland. 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks