Natriumoxybaat

Active substance
Natriumoxybaat
Domain
Neurological disorders
Reason of inclusion in Horizonscan Geneesmiddelen
Indication extension
Main indication
Sleep disorders
Extended indication
Narcolepsy with cataplexy in children (ages 7-17)

1. Product

Proprietary name
Xyrem
Manufacturer
UCB
Mechanism of action
Receptor agonist
Route of administration
Oral
Therapeutical formulation
Drink
Budgetting framework
Extramural (GVS)
Additional comments
Central-nervous-system depressant.

2. Registration

Registration route
Centralised (EMA)
Submission date
August 2018
Expected Registration
August 2020
Orphan drug
No
Registration phase
Registration application pending
Additional comments
In juni 2018 presenteerde de fabrikant positieve langetermijnresultaten van de EXPRESS studie.

3. Therapeutic value

Therapeutic value
No judgement
Substantiation
Studie toont positieve resultaten met vergelijkbaar veiligheidsprofiel met volwassenen.
Frequency of administration
1 times a day
Dosage per administration
≤9 g/nacht
References
Farmacotherapeutisch kompas; NCT02221869; Plazzi et al. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494.

4. Expected patient volume per year

Patient volume

< 60

Market share is generally not included unless otherwise stated.

References
CBS; GIPdatabank
Additional comments
In 2016 gebruikten 406 mensen Xyrem. In 2017 waren er 2.162.869 inwoners in Nederland van de leeftijd 7-17 en 13.677.409 van de leeftijd >17. Als de populatie >17 in totaal 406 gebruikers telde dan zou dit voor 7-17 betekenen dat er 64 gebruikers zijn.

5. Expected cost per patient per year

Cost
8,000
References
GIPdatabank
Additional comments
In 2016 werd €8.108 vergoed per gebruiker van Xyrem.

6. Potential total cost per year

Total cost

480,000

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
Fibromyalgia
References
Clinicaltrials.gov

9. Other information

There is currently no futher information available.