SPINRAZA (nusinersen), antisense oligonucleotide

High clinical benefit for 5q spinal muscular atrophy types I and II and moderate clinical added value for the therapeutic strategy.

High clinical benefit for 5q spinal muscular atrophy type III but no proven clinical added value for the therapeutic strategy.

Insufficient clinical benefit to justify reimbursement for 5q spinal muscular atrophy type IV.

• SPINRAZA has been granted a marketing authorisation for the aetiological treatment of 5q spinal muscular atrophy.

•  In 5q spinal muscular atrophy types I and II, its superiority compared to a sham injection procedure has been demonstrated on motor function improvement criteria, assessed on validated scales in two clinical studies, and in terms of survival without ventilation and survival rate in one of the 2 studies.

• In 5q spinal muscular atrophy type III, the data from 2 open-label phase II studies are not sufficient to reach a conclusion in respect of its efficacy, however, a benefit could be expected for some patients.

• The effect of nusinersen has not been studied in patients with 5q spinal muscular atrophy type IV.

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