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Dossier assessment

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

Status
Status: Commission completed
Project no.
A21-68
Last updated

Report documents

  1. Extract of dossier assessment

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

    published on
    Document type
    PDF-Dokument, 508 kB, EN, PDF
    Download file : Extract of dossier assessment (PDF-Dokument, 508 kB, EN, PDF)

  2. Dossier assessment

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

    published on
    Document type
    PDF-Dokument, 921 kB, DE, PDF
    Download file : Dossier assessment (PDF-Dokument, 921 kB, DE, PDF)

Dossier assessment

Indication:

Patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene

Result of dossier assessment:
  • SMA type 1: added benefit not proven.
  • SMA type 2: added benefit not proven.
  • SMA type 3: added benefit not proven.
  • Presymptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene: added benefit not proven.

Press releases

Spinal muscular atrophy: second and third drug have undergone benefit assessment

There is a hint of an added benefit of risdiplam for SMA type 1; no added benefit of onasemnogene abeparvovec has been proven for any type. Different exclusion criteria of the studies make comparisons difficult.
Press release

Additional information

Federal Joint Committee (G-BA)

2021-11-04 A G-BA decision was published.

G-BA documents on this decision

Project details

Project no.
A21-68
Commission
Commission awarded on 17.05.2021 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. Addendum

    Onasemnogene abeparvovec (spinal muscular atrophy) - Addendum to Commission A21-68

    Project no.
    A21-128
    Last updated

    Status: Commission completed

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press@iqwig.de

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