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Natriumoxybaat

Xyrem

Extended indication Treatment of narcolepsy with cataplexy in adult patients, adolescents and children from the age of 7
Therapeutic value No judgement
Total cost 480,000.00
Registration phase Positive CHMP opinion

Product

Active substance Natriumoxybaat
Domain Neurological disorders
Reason of inclusion Indication extension
Main indication Sleep disorders
Extended indication Treatment of narcolepsy with cataplexy in adult patients, adolescents and children from the age of 7 years.
Proprietary name Xyrem
Manufacturer UCB
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Drink
Budgetting framework Extramural (GVS)
Additional remarks Central-nervous-system depressant.

Registration

Registration route Centralised (EMA)
Submission date August 2018
Expected Registration January 2021
Orphan drug No
Registration phase Positive CHMP opinion
Additional remarks In juni 2018 presenteerde de fabrikant positieve langetermijnresultaten van de EXPRESS studie. Positieve CHMP-opinie in november 2020.

Therapeutic value

Therapeutic value No judgement
Substantiation Studie toont positieve resultaten met vergelijkbaar veiligheidsprofiel met volwassenen.
Duration of treatment continuous
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.

Expected patient volume per year

Patient volume

< 60

Market share is generally not included unless otherwise stated.
References CBS; GIPdatabank
Additional remarks In 2016 gebruikten 406 mensen Xyrem. In 2017 waren er 2.162.869 inwoners in Nederland van de leeftijd 7-17 jaar en 13.677.409 van de leeftijd >17 jaar. Als de populatie >17 jaar in totaal 406 gebruikers telde dan zou dit voor 7-17 jaar betekenen dat er 64 gebruikers zijn.

Expected cost per patient per year

Cost 8,000.00
References GIPdatabank
Additional remarks In 2016 werd €8.108 vergoed per gebruiker van Xyrem.

Potential total cost per year

Total cost

480,000.00

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 Fibromyalgia
References Clinicaltrials.gov

Other information

There is currently no futher information available.