Dossier assessment
Risdiplam (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V
Report documents
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Extract of dossier assessment
Risdiplam (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V
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Dossier assessment
Risdiplam (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V
Dossier assessment
Indication:
Patients with 5q spinal muscular atrophy (SMA)
Result of dossier assessment:
- Patients 2 months of age and older with SMA type 1: hint of a non-quantifiable added benefit.
- Patients 2 months of age and older with SMA type 2 or 3, and pre-symptomatic patients with up to 4 SMN2 gene copies: 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.
Additional information
Federal Joint Committee (G-BA)
2021-10-21: A G-BA decision was published.
Project details
- Project no.
- A21-50
- Commission
- Commission awarded on 30.04.2021 by the Federal Joint Committee (GBA).
- Report type
- Dossier assessment
- Status
- Commission completed
- Department/Division
- Drug Assessment
- Topic
- Head and nerves
- Note
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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
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Addendum
Risdiplam (spinal muscular atrophy) - Addendum to Commission A21-50
Status: Commission completed