Natriumoxybaat Medicine 13 December 2018 Extended indication Narcolepsy with cataplexy in children (ages 7-17) Therapeutic value No judgement Total cost € 480,000.00 Registration phase Registration application pending Product Active substance Natriumoxybaat Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Sleep disorders Extended indication Narcolepsy with cataplexy in children (ages 7-17) Current proprietary name Already available biosimilars / generics Proprietary name Xyrem Manufacturer UCB Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Drink Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Central-nervous-system depressant. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date August 2018 Expected Registration August 2020 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks In juni 2018 presenteerde de fabrikant positieve langetermijnresultaten van de EXPRESS studie. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Studie toont positieve resultaten met vergelijkbaar veiligheidsprofiel met volwassenen. Duration of treatment 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. Additional remarks Expected patient volume per year Patient volume < 60 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 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. Expected cost per patient per year Cost € 8,000.00 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. References GIPdatabank Additional remarks In 2016 werd €8.108 vergoed per gebruiker van Xyrem. Potential total cost per year Total cost € 480,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Fibromyalgia References Clinicaltrials.gov Additional remarks Other information Additional remarks