Eculizumab Medicine 06 June 2023 Extended indication Extension of indication to include treatment of paediatric patients aged 6 to less than 18 years with acetylcholine receptor-antibody (AChR-Ab) positive (+) refractory gMG. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Eculizumab Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Muscular diseases other Extended indication Extension of indication to include treatment of paediatric patients aged 6 to less than 18 years with acetylcholine receptor-antibody (AChR-Ab) positive (+) refractory gMG. Current proprietary name Already available biosimilars / generics Proprietary name Soliris Manufacturer Alexion Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date February 2023 Expected Registration November 2023 Orphan drug Yes 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 Duration of treatment 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 1200 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 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.spierziekten.nl/fileadmin/user_upload/VSN/documenten/Huisartsenbrochures/M010-MG-habrochmetbrieven.pdf Additional remarks De prevalentie is 1 patiënt per 10.000 inwoners. Expected cost per patient per year Cost References Additional remarks Voor alle indicaties van eculizumab geldt een financieel arrangement lopend tot en met 31 december 2025 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