Extended indication To prevent or delay the onset of type 1 diabetes (T1D) in relatives determined to be at very high ri
Therapeutic value Possibly no place in the treatment regimen
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.
Manufacturer Provention bio
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments CD3 antigen inhibitor

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Particularity New medicine with Priority Medicines (PRIME)
Submission date 2021
Expected Registration 2022
Orphan drug No
Registration phase Clinical trials

Therapeutic value

Therapeutic value Possibly no place in the treatment regimen
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
References NCT01030861
Additional comments 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

References GIPdatabank;
Additional comments 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 tweedegraads 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No

Other information

There is currently no futher information available.