Tasimelteon Medicine 03 December 2024 Extended indication Extension of indication to include the treatment of nighttime sleep disturbances in adults with Smith Magenis Syndrome (SMS). Therapeutic value No estimate possible yet Total cost Registration phase Negative CHMP opinion Product Active substance Tasimelteon Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Sleep disorders Extended indication Extension of indication to include the treatment of nighttime sleep disturbances in adults with Smith Magenis Syndrome (SMS). Current proprietary name Already available biosimilars / generics Proprietary name Hetlioz Manufacturer Vanda Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Melatoninereceptoragonist Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date October 2024 Expected Registration Orphan drug Yes Registration phase Negative CHMP opinion Reimbursement Medicine sluice Additional remarks Negatieve CHMP-opinie in november 2025. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er is geen behandeling voor Smith Magenis Syndrome. Er zijn nog geen resultaten beschikbaar om een inschatting over de therapeutische waarde op te baseren. Duration of treatment Frequency of administration 1 times a day Dosage per administration 20mg References NCT02231008 (VP-VEC-162-2401) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie Additional remarks SMS is een zeldzame aandoening die voorkomt bij ongeveer 1 op de 15.000 tot 25.000 pasgeborenen. Dat is ongeveer zeven tot tien kinderen per jaar. De registratie is voor volwassenen. Expected cost per patient per year Cost € 400.00 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 GoodRx Additional remarks In de Verenigde Staten is de prijs voor tasimelteon ongeveer $14.000 voor 30 capsules van 20mg en dus $467 per capsule. In principe is de dosering 1 capsule. Prijs per patiënt per jaar dus sterk afhankelijk van de duur van de behandeling. Tasimelteon wordt op dit moment niet vergoed in Nederland. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks