Mexiletine HCl Medicine 09 January 2018 Extended indication Symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders. Therapeutic value No judgement Total cost € 24,000,000.00 Registration phase Registered Product Active substance Mexiletine HCl Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Muscular diseases other Extended indication Symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders. Current proprietary name Already available biosimilars / generics Proprietary name Namuscla Manufacturer Lupin Portfolio holder Mechanism of action Ion channel blocker Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Toedieningsvorm: naast capsules komt mexiletine HCl ook als oplossing beschikbaar (bron EMA). Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2017 Expected Registration December 2018 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Mexiletine HCl is al op de markt in Frankrijk. Positieve CHMP-opinie oktober 2018. Geregistreerd in december 2018. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 3 times a day Dosage per administration 150 mg References NCT01406873 Additional remarks Expected patient volume per year Patient volume < 300 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.radboudumc.nl/nieuws/2019/radboudumc-bezorgd-over-toegankelijkheid-medicijn Additional remarks Er lijden in Nederland ongeveer 300 patiënten aan niet-dystrofische myotonie Expected cost per patient per year Cost € 80,000.00 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 https://www.radboudumc.nl/nieuws/2019/radboudumc-bezorgd-over-toegankelijkheid-medicijn Additional remarks In Duitsland en het Verenigd Koninkrijk kost een behandeljaar €80.000 Potential total cost per year Total cost € 24,000,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References SPS Additional remarks Other information Additional remarks