Belimumab Medicine 03 December 2024 Extended indication Extension of indication to include treatment of paediatric patients from 5 years of age with active, autoantibody-positive systemic lupus erythematosus (SLE) for BENLYSTA, based on final results from study 200908; this is a worldwide population pharmacokinetic analysis of subcutaneous administered belimumab plus standard therapy to pediatric patients aged 5-17 years with systematic lupus erythematous (SLE), which was aimed to describe the pharmacokinetic (PK) analysis of belimumab to support an appropriate weight-based dosing regimen for subcutaneous administration in paediatric patients aged 5-17 years with SLE Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Belimumab Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Other chronic immune diseases Extended indication Extension of indication to include treatment of paediatric patients from 5 years of age with active, autoantibody-positive systemic lupus erythematosus (SLE) for BENLYSTA, based on final results from study 200908; this is a worldwide population pharmacokinetic analysis of subcutaneous administered belimumab plus standard therapy to pediatric patients aged 5-17 years with systematic lupus erythematous (SLE), which was aimed to describe the pharmacokinetic (PK) analysis of belimumab to support an appropriate weight-based dosing regimen for subcutaneous administration in paediatric patients aged 5-17 years with SLE Current proprietary name Already available biosimilars / generics Proprietary name Benlysta Manufacturer GSK Portfolio holder Mechanism of action Immunostimulation Route of administration Subcutaneous Therapeutical formulation Unknown Budgetting framework Unknown Centre of expertise Additional remarks Monoclonaal antilichaam gericht tegen B-lymphocyte stimulator (BLyS). Dit betreft de goedkeuring van de subcutane toedieningsweg voor patiënten tussen de 5 tot 17 jaar met SLE in een 200mg auto injector. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date July 2024 Expected Registration May 2025 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Belimumab wordt momenteel niet veel ingezet bij volwassen patiënten. Belimumab is niet geschikt voor de behandeling van ernstige SLE en ook niet bij de standaard behandeling. Dit geneesmiddel heeft alleen meerwaarde bij patiënten die hier tussenin vallen. De verwachting van kinderreumatologen is dat sommige patiënten die een indicatie voor belimumab initra veneus hebben eventueel over gaan op subcutane toediening gezien het gebruiksgemak en het thuisbehandeling mogelijk maakt. Gezien de kleine patiëntaantallen is er geen grote budgetimpact te verwachten. Duration of treatment Frequency of administration Dosage per administration References NCT04179032 (200908) Additional remarks Expected patient volume per year Patient volume 20 - 40 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Op basis van inschatting Forigerimod (Rigerimod) / Belimumab Additional remarks In de beoordeling van belimumab heeft het Zorginstituut gerekend met: 'De prevalentie van SLE is 28 tot 40 per 100.000 inwoners (ongeveer 4.470 patiënten in Nederland). Ongeveer 8% van de patiënten met SLE heeft een hoge mate van ziekteactiviteit, ondanks een standaardbehandeling (zo’n 357 patiënten).' Gezien het hier alleen 5 tot 18 jarigen betreft is de inschatting dat zo'n 5 tot 10% van deze populatie in aanmerking zou komen voor belimumab bij deze indicatie. 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 Unknown Indication extensions References Additional remarks Other information Additional remarks