[A21-68] Onasemnogene abeparvovec (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V
Last updated 04.11.2021
Commission awarded on 17.05.2021 by the Federal Joint Committee (G-BA).
Muscles, bones and joints
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
- 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.
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.