SPINRAZA (nusinersen) - Spinal muscular atrophy

Opinions on drugs - Posted on Jul 10 2024

Reason for request

Reassessment

Summary of opinion

Favourable opinion for reimbursement:

“in pre-symptomatic paediatric patients with genetically diagnosed 5q spinal muscular
atrophy with 2 to 3 copies of the SMN2 gene,
5q spinal muscular atrophy types 1, 2 or 3”

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of SPINRAZA (nusinersen) 12 mg solution for injection remains substantial in the indications:

“5q spinal muscular atrophy type 1, 2 or 3.
in pre-symptomatic paediatric patients with genetically diagnosed 5q spinal muscular atrophy with 2 to 3 copies of the SMN2 gene”.

Clinical Added Value

moderate

Pre-symptomatic paediatric patients with genetically diagnosed 5q spinal muscular atrophy with 2 to 3 copies of the SMN2 gene.

The Committee considers that SPINRAZA (nusinersen) provides a moderate clinical added value (CAV III) in the care pathway, in the same way as ZOLGENSMA (onasemnogene abeparvovec).

Patients with 5q spinal muscular atrophy type 1.

The Committee considers that SPINRAZA (nusinersen) provides a moderate clinical added value (CAV III) in the care pathway, in the same way as ZOLGENSMA (onasemnogene abeparvovec) and EVRYSDI (risdiplam).

Patients with 5q spinal muscular atrophy type 2.

The Committee considers that SPINRAZA (nusinersen) provides a moderate clinical added value (CAV III) in the care pathway, in the same way as EVRYSDI (risdiplam).

Patients with 5q spinal muscular atrophy type 3.

The Committee considers that SPINRAZA (nusinersen) provides no clinical added value (CAV V) in the care pathway.

no clinical added value