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Vestronidase alfa

Mepsevii

Extended indication Mucopolysaccharidosis VII (MPS VII - Sly disease)
Therapeutic value No judgement
Total cost 700,000.00

Product

Active substance Vestronidase alfa
Domain Metabolism and Endocrinology
Main indication Metabolic diseases
Extended indication Mucopolysaccharidosis VII (MPS VII - Sly disease)
Proprietary name Mepsevii
Manufacturer Ultragenyx
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date June 2017
Expected Registration April 2018
Orphan drug Yes
Additional remarks In US fast track bij FDA

Therapeutic value

Current treatment options Supportive care. Er is geen specifieke behandeling voor Sly Disease.
Therapeutic value No judgement
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 2 times a month
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

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).

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

700,000.00

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.