Cannabidiol

Active substance
Cannabidiol
Domain
Neurological disorders
Reason of inclusion in Horizonscan Geneesmiddelen
Indication extension
Main indication
Epilepsy
Extended indication
Seizures in Tuberous Sclerosis Complex.

1. Product

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

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
2020
Expected Registration
2020
Orphan drug
Yes
Registration phase
Clinical trials

3. Therapeutic value

Current treatment options
Anti-epileptica
Therapeutic value
No judgement
Substantiation
De therapeutische waarde lijkt op basis van de fase 3 studie aanzienlijk, echter is de invloed van de geneesmiddel-interactie 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.
Duration of treatment
Not found
Frequency of administration
2 times a day
Dosage per administration
25-50 mg/kg/dag
References
NCT02544763

4. 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 comments
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.

5. Expected cost per patient per year

Cost
29,000
References
FiercePharma
Additional comments
In de Verenigde Staten is de lijstprijs gesteld op $32.500 (is omgerekend ongeveer €29.000).

6. Potential total cost per year

Total cost

27,405,000

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.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

Indication extension
Yes
Indication extensions
Rett syndroom
References
Website GW pharmaceuticals

9. Other information

There is currently no futher information available.