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 Other muscular diseases
Extended indication Symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders.
Proprietary name Namuscla
Manufacturer Lupin
Mechanism of action Ion channel blocker
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional comments Toedieningsvorm: naast capsules komt mexiletine HCl ook als oplossing beschikbaar (bron EMA).

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date September 2017
Expected Registration December 2018
Orphan drug Yes
Registration phase Registered
Additional comments Mexiletine HCl is al op de markt in Frankrijk. Positieve CHMP-opinie oktober 2018. Geregistreerd in december 2018. ZIN beoordeelt of mexiletine opgenomen kan worden in het GVS, start beoordeling juli 2020.

Therapeutic value

Therapeutic value No judgement
Duration of treatment continuous
Frequency of administration 3 times a day
Dosage per administration 150 mg
References NCT01406873

Expected patient volume per year

Patient volume

< 300

Market share is generally not included unless otherwise stated.

References https://www.radboudumc.nl/nieuws/2019/radboudumc-bezorgd-over-toegankelijkheid-medicijn.
Additional comments Er lijden in Nederland ongeveer 300 patiënten aan niet-dystrofische myotonie.

Expected cost per patient per year

Cost 80,000.00
References https://www.radboudumc.nl/nieuws/2019/radboudumc-bezorgd-over-toegankelijkheid-medicijn.
Additional comments In Duitsland en het Verenigd Koninkrijk kost een behandeljaar €80.000.

Potential total cost per year

Total cost

24,000,000.00

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

There is currently nothing known about off label use.

Indication extension

Indication extension No
References SPS

Other information

There is currently no futher information available.