Expand all sections

Eculizumab

Soliris

Extended indication Extension of indication to include treatment of paediatric patients aged 6 years and above with acet
Therapeutic value No estimate possible yet
Registration phase Registered

Product

Active substance Eculizumab
Domain Neurological disorders
Reason of inclusion Indication extension
Main indication Muscular diseases other
Extended indication Extension of indication to include treatment of paediatric patients aged 6 years and above with acetylcholine receptor-antibody (AChR-Ab) positive (+) refractory gMG.
Proprietary name Soliris
Manufacturer Alexion
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date November 2022
Expected Registration July 2023
Orphan drug Yes
Registration phase Registered
Additional remarks Positieve CHMP-opinie juni 2023

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation De fase 3 studie bij pediatrische patiënten met refractaire myasthenia gravis is nog niet officieel gepubliceerd. In de klinische trial (NCT03759366) worden 11 kinderen, in de leeftijd 6 tot 17, met refractaire en AChR-positieve gMG behandeld met Soliris voor 26 weken. De resultaten laten zien dat de gemiddelde score op de Quantitative MG (QMG) schaal, die de mate van MG meet, met 5,8 punten toenam. Dit toont aan dat ernstige symptomen van de ziekte verminderen na gebruik.
Duration of treatment continuous
Frequency of administration 1 times every 2 weeks
Dosage per administration 300 - 1200mg
References NCT03759366
Additional remarks Eculizumab will be administered at doses of 300, 600, 900, or 1,200 milligrams (mg), based on the participant's current body weight. In the Primary Evaluation Treatment Period (26 weeks), eculizumab will be administered weekly during the initial induction phase and every 2 weeks during the maintenance phase.

Expected patient volume per year

Patient volume

3

Market share is generally not included unless otherwise stated.
References Lanet Neurol. 2022. Juvenile myasthenia gravis: a nationwide study in Norway suggested an average annual incidence of 1·6 cases per 1 million people and a prevalence of 3·6–13·8 cases per 1 million people, with more girls affected than boys (1). Expertopinie(2);
Additional remarks In Nederland zijn geschat 3.000 patiënten met myasthenia gravis (167 per miljoen) , de helft hiervan gebruikt immunosuppressiva (rond de1.500) en bij 10 tot 20% hiervan wordt wegens problemen met chronische stabiliteit derdelijns medicatie overwogen waaronder complementremmers (150 tot 300) (1). Hierbij zullen slechts enkele kinderen zijn. Het zou om totaal 30 kinderen in Nederland gaan en als daarvan 10% complementremmers nodig heeft gaat het om hooguit 3 patiënten (2).

Expected cost per patient per year

Additional remarks Voor alle indicaties van eculizumab geldt een financieel arrangement lopend tot en met 31 december 2025.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.