Expand all sections

Vestronidase alfa

Mepsevii

Extended indication Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis
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).
Proprietary name Mepsevii
Manufacturer Ultragenyx
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
Submission date June 2017
Expected Registration August 2018
Orphan drug Yes
Registration phase Registered
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
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 continuous
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.
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
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

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.

Off label use

Off label use No

Indication extension

Indication extension No

Other information

There is currently no futher information available.