Vestronidase alfa Medicine 09 January 2018 Extended indication Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). Therapeutic value No judgement yet Total cost € 1,050,000.00 Registration phase Registered Product Active substance Vestronidase alfa Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). Current proprietary name Already available biosimilars / generics Proprietary name Mepsevii Manufacturer Ultragenyx Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise ErasmusMC Additional remarks Enzyme Replacement Therapy (ERT): recombinant form of human beta-glucuronidase (rhGUS) Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date June 2017 Expected Registration August 2018 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks In US fast track bij FDA. Positieve CHMP-opinie in juli 2018. Gergeistreerd in augustus 2018. Therapeutic value Current treatment options Supportive care. Symptoombestrijding. Er is geen specifieke behandeling voor Sly Disease. Therapeutic value No judgement yet This assessment does not indicate any potential inclusion in the package. Substantiation MPS VII (ziekte van SLy) is een uiterst zeldzame lysosomale stapelingsziekte veroorzaakt door deficientie van beta-glucuronidase. Hierdoor ontstaat stapeling van glycosaminoglycanen (dermatan- heparan- en chondroitinesulfaat). Een case report met dosering 2 mg/kg laat verbetering zien mbt lever en miltvolume, afname GAG's in urine en mgl longfunctie. Trials: 'In EU, the primary endpoint is the percent reduction in urinary glycosaminoglycans (GAG) excretion after 24 weeks of treatment. The EMA indicated that a trend in improvement in clinical endpoints would also be necessary for approval.' Duration of treatment Frequency of administration 1 times every 2 weeks Dosage per administration 4 mg/kg References Fox et al. Mol Genet Metab. 2015 February ; 114(2): 203–208; Specialist Pharmacy Service; clinicaltrials.gov Additional remarks Toediening om de week. Expected patient volume per year Patient volume 2 - 4 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Zeer zeldzame aandoening. Wereldwijde geboorteprevalentie is <1:250.000. Aantal patienten in Nederland is zeer laag (2 bekend in ErasmusMC). De fabrikant geeft aan dat er momenteel 4 gediagnosticeerde patiënten in Nederland in leven zijn. Expected cost per patient per year Cost € 200,000.00 - 500,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Additional remarks Vergelijkbaar met andere enzymtherapieen. Totale kosten van ERT, aangezien er geen alternatief is. Potential total cost per year Total cost € 1,050,000.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks