Nusinersen Medicine 09 January 2018 Extended indication Behandeling van 5q spinale spieratrofie (spinal muscular atrophy, SMA). Therapeutic value No judgement Total cost € 764,910,000.00 Registration phase Unknown Product Active substance Nusinersen Domain Neurological disorders Reason of inclusion Main indication Muscular diseases Extended indication Behandeling van 5q spinale spieratrofie (spinal muscular atrophy, SMA). Current proprietary name Already available biosimilars / generics Proprietary name Spinraza Manufacturer Biogen Portfolio holder Mechanism of action Unknown Route of administration Intrathecal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP Unknown Submission date November 2016 Expected Registration May 2017 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Spinraza geregistreerd bij de EMA voor SMA. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 2,833 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.erfelijkheid.nl/ziektes/spinale-musculaire-atrofie-sma Additional remarks Ongeveer 3 tot 5 op de 30.000 mensen hebben spinale musculaire atrofie. Daarvan komt SMA type 1 het meest voor, ongeveer 50 tot 60%. Van de mensen met SMA heeft ongeveer 30 % (1/3e) type 2. Type 3 en 4 komen heel weinig voor. Expected cost per patient per year Cost € 270,000.00 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 Prescribing Outlook 2017, Fabrikant, GIP. Additional remarks Prescribing Outlook 2017: 'Published annual cost at list price in launch EU countries is up to €270,000 per patient/year for three-dose maintenance therapy; it may be higher in the first year due to the initial loading dose regimen.' Fabrikant geeft een range aan van €210.000-270.000 p.p.p.j. Potential total cost per year Total cost € 764,910,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks