[A20-114] Nusinersen (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V

Last updated 20.05.2021

Project no.:
A20-114

Commission:
Commission awarded on 02.12.2020 by the Federal Joint Committee (G-BA).

Report type:
Dossier assessment

Status:
Commission completed

Department/Division:
Drug Assessment

Application field:
Muscles, bones and joints

Indication:

Patients with 5q spinal muscular atrophy (SMA)

Result of dossier assessment:

Early onset of disease (infantile form, type 1): indication of major added benefit. Later onset of disease (type 2, type 3 and type 4): added benefit not proven. Pre-symptomatic patients: hint of non-quantifiable added benefit.

Note:

After completion of the assessment by IQWiG the Federal Joint Committee (G-BA) conducts a commenting procedure. This may provide supplementary information and as a result lead to a modified benefit assessment. Further information and the decision on the early benefit assessment can be found on the relevant page of the G-BA website.

01.03.2021

Nusinersen in SMA: indication of major added benefit in children with early-onset disease

Treatment delays permanent ventilation of the children and prolongs life. In addition, the children develop more motor skills.

Federal Joint Committee (G-BA)

2021.05.20 A G-BA decision was published.

G-BA documents on this decision

Contact

Info Service

Subscribe to daily updated information on projects, calls for tender, and events as well as our press releases.

Contact form

Questions about commissions, publications and press releases can be sent to us via this form.