Nusinersen

final resolution

2021-05-20

Arzneimittel-Richtlinie/Anlage XII: Nusinersen (Neubewertung eines Orphan Drugs nach Überschreitung der 50 Mio. Euro-Grenze: Spinale Muskelatrophie)

Evidence-checked summary

The G-BA found an indication of major added benefit for Patienten mit 5q-assoziierter spinaler Muskelatrophie (5q-SMA) Typ 1:. The appropriate comparator was BSC. The G-BA found a hint of considerable added benefit for Patienten mit 5q-SMA Typ 2. The appropriate comparator was BSC. The G-BA found no proven added benefit for Patienten mit 5q-SMA Typ 3 / 4. The appropriate comparator was BSC. The G-BA found a hint of major added benefit for präsymptomatische Patienten mit 5q-SMA und 2 SMN2-Genkopien. The appropriate comparator was BSC. The G-BA found a hint of non-quantifiable added benefit for präsymptomatische Patienten mit 5q-SMA und 3 SMN2-Genkopien. The appropriate comparator was BSC. The G-BA found no proven added benefit for präsymptomatische Patienten mit 5q-SMA und mehr als 3 SMN2-Genkopien. The appropriate comparator was BSC. The G-BA issued a final resolution on 2021-05-20. The official page states that it took effect as follows: 20.05.2021. The page lists Arzneimittel-Richtlinie as the normative target.

This is the latest validated public action attached to this case. It is separate from the IQWiG assessment result.