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Dossier assessment

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

Status
Status: Commission completed
Project no.
A20-114
Last updated

Report documents

  1. Extract of dossier assessment

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

    published on
    Document type
    PDF-Dokument, 989 kB, EN, PDF
    Download file : Extract of dossier assessment (PDF-Dokument, 989 kB, EN, PDF)

  2. Dossier assessment

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

    published on
    Document type
    PDF-Dokument, 1 MB, DE, PDF
    Download file : Dossier assessment (PDF-Dokument, 1 MB, DE, PDF)

Dossier assessment

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.

Press releases

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.
Press release

Additional information

Federal Joint Committee (G-BA)

2021.05.20 A G-BA decision was published.

G-BA documents on this decision

Project details

Project no.
A20-114
Commission
Commission awarded on 02.12.2020 by the Federal Joint Committee (GBA).
Report type
Dossier assessment
Status
Commission completed
Department/Division
Drug Assessment
Topic
Muscles, bones and joints
Note

After completion of the assessment by IQWiG the Federal Joint Committee (GBA) 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 GBA website.

Connected Projects

  1. Dossier assessment

    Nusinersen (spinal muscular atrophy) - Assessment according to §35a (para. 1., sentence 10) Social Code Book V

    Project no.
    G17-05
    Last updated

    Status: Commission completed

  2. Addendum

    Nusinersen (spinal muscular atrophy) - Addendum to Commission A20-114

    Project no.
    A21-43
    Last updated

    Status: Commission completed

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