Extended indication Behandeling van 5q spinale spieratrofie (spinal muscular atrophy, SMA).
Therapeutic value No judgement
Total cost 764,910,000.00

Product

Active substance Nusinersen
Domain Neurological disorders
Main indication Muscular diseases
Extended indication Behandeling van 5q spinale spieratrofie (spinal muscular atrophy, SMA).
Proprietary name Spinraza
Manufacturer Biogen
Route of administration Intrathecal
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Submission date November 2016
Expected Registration May 2017
Orphan drug Yes
Additional remarks Spinraza geregistreerd bij de EMA voor SMA.

Therapeutic value

Therapeutic value No judgement
Duration of treatment Not found

Expected patient volume per year

Patient volume

< 2,833

Market share is generally not included unless otherwise stated.

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
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

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.