[A21-68] Onasemnogene abeparvovec (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V

Last updated 04.11.2021

Project no.:
A21-68

Commission:
Commission awarded on 17.05.2021 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) 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.

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.

16.08.2021

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. Differe...

Project no. Title Status
A21-128 Onasemnogene abeparvovec (spinal muscular atrophy) - Addendum to Commission A21-68 Commission completed

Federal Joint Committee (G-BA)

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

G-BA documents on this decision

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