[A21-43] Nusinersen (spinal muscular atrophy) - Addendum to Commission A20-114

Last updated 20.05.2021

Project no.:
A21-43

Commission:
Commission awarded on 07.04.2021 by the Federal Joint Committee (G-BA).

Report type:
Addendum

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:

Patients with early onset of disease (infantile form, SMA type 1): indication of major added benefit.

Patients with later onset of disease (SMA type 2, type 3 and type 4): added benefit not proven.

Pre-symptomatic patients with 5q SMA: hint of non-quantifiable added benefit.

Note:

If the need for additional work on a project commissioned by the G-BA arises during consultations, then IQWiG presents a report in the form of an "addendum". The G-BA subsequently decides on the extent of the added benefit, thus completing the early benefit assessment.

Federal Joint Committee (G-BA)

2021.05.20 A G-BA decision was published.

G-BA documents on this decision

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