Expand all sections

Cannabidiol

Epidyolex

Extended indication Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis
Therapeutic value No estimate possible yet
Total cost 27,405,000.00
Registration phase Registered

Product

Active substance Cannabidiol
Domain Neurological disorders
Reason of inclusion Indication extension
Main indication Epilepsy
Extended indication Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older.
Proprietary name Epidyolex
Manufacturer GW Pharmaceuticals
Mechanism of action Cannabinoid
Route of administration Oral
Therapeutical formulation Solution
Budgetting framework Extramural (GVS)
Centre of expertise A small-molecule cannabinoid compound

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date April 2020
Expected Registration April 2021
Orphan drug Yes
Registration phase Registered
Additional remarks Positieve CHMP-opinie in februari 2021.

Therapeutic value

Current treatment options Anti-epileptica
Therapeutic value No estimate possible yet
Substantiation De therapeutische waarde lijkt op basis van de fase 3 studie aanzienlijk, echter is de invloed van de geneesmiddelinteractie mogelijk zorgelijk. Cannabidiol remt onder andere CYP3A4 en CYP2C9, wat respectievelijk clobazam en valproaat metaboliseert. Deze middelen zijn in de fase 3 trial door 45% en 27% van de patiënten gebruikt.
Frequency of administration 2 times a day
Dosage per administration 25-50 mg/kg/dag
References NCT02544763

Expected patient volume per year

Patient volume

559 - 1,331

Market share is generally not included unless otherwise stated.
References Brochure Tubereuze Sclerose Complex, NHG 2014 Chu-Shore, C., Major, P., Camposano, S., Muzykewicz, D., & Thiele, E. (2009). The natural history of epilepsy in tuberous sclerosis complex. Epilepsia, 51(7), 1236-1241.
Additional remarks Naar schatting zijn er in Nederland ongeveer 1.500-2.500 patiënten met TSC. Ongeveer 70%-80% hiervan vertoont epileptische aanvallen (1.050-2.000). Alleen patiënten met refractaire epilepsie zullen in aanmerking komen voor deze behandeling. "85.2% had a "history of seizure'' en ''Of epilepsy patients, 62,5% developed refractory epilepsy'' (Chu-Shore et al, 2009). Dit komt neer op 85,2% van 1.050-2.500 is 895-2.130, waarvan 62,5% is een totaal van 559-1.331 patiënten.

Expected cost per patient per year

Cost 29,000.00
References FiercePharma
Additional remarks In de Verenigde Staten is de lijstprijs gesteld op $32.500 (is omgerekend ongeveer €29.000).

Potential total cost per year

Total cost

27,405,000.00

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 Yes
Indication extensions Rett syndroom
References Website GW pharmaceuticals

Other information

There is currently no futher information available.