Nusinersen

(Spinraza®)

Nusinersen

Drug updated on 6/24/2024

Dosage FormInjection (intrathecal; 12 mg/5 mL)
Drug ClassSurvival motor neuron-2 (SMN2)-directed antisense oligonucleotides
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Latest News

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Summary
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  • Nusinersen (Spinraza) is indicated for the treatment of spinal muscular atrophy (SMA) in both pediatric and adult patients, offering a significant profile of efficacy and safety across a wide range of individuals.
  • The analysis stemmed from nine systematic reviews or meta-analyses regarding Spinraza's role in SMA treatment, comparing it to other treatments such as risdiplam, onasemnogene abeparvovec, and more traditional approaches.
  • When compared with risdiplam for improving motor function in SMA patients, nusinersen demonstrates considerable benefits across various motor function scales without a significant increase in adverse events. Both treatments exhibit similar rates of adverse events, suggesting favorable safety profiles but with potentially higher efficacy for nusinersen.
  • Specifically, in children and adolescents, the studies suggest rare direct adverse events from nusinersen while effectively minimizing common serious fatal adverse events, presenting strong evidence for its safety and effectiveness within this younger demographic.
  • For adults with an extended duration of the disease, as assessed by one study, statistically significant improvements in motor functions were observed after using nusinersen, with minimal serious side effects, underlining its effectiveness for older patient groups.
  • Compared to historical treatments such as creatine, phenylbutyrate, gabapentin, thyrotropin-releasing hormone, hydroxyurea, combination therapy with valproate, acetyl-L-carnitine, none demonstrated statistically significant effects, highlighting the deficiency of older drugs against SMA types II and III. This emphasizes the breakthrough status of newer therapies like nusinersen.

Product Monograph / Prescribing Information

Document TitleYearSource
Spinraza (nusinersen) Prescribing Information.2023Biogen, Cambridge, MA

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Safety and efficacy of nusinersen and risdiplam for spinal muscular atrophy: a systematic review and meta-analysis of randomized controlled trials.2023Brain Sciences
Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: a systematic review and meta-analysis.2022Frontiers in Pediatrics
Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: a systematic review of real-world study data2022European Journal of Paediatric Neurology
Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: a systematic review of real-world study data.2022Medicina
Nusinersen in adults with 5q spinal muscular atrophy: a systematic review and meta-analysis.2022Neurotherapeutics
How does risdiplam compare with other treatments for types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.2022Journal of Comparative Effectiveness Research
Motor function in type 2 and 3 SMA patients treated with nusinersen: a critical review and meta-analysis.2021Orphanet Journal of Rare Diseases
Nusinersen treatment of spinal muscular atrophy - a systematic review.2020Danish Medical Journal
Drug treatment for spinal muscular atrophy types II and III.2020The Cochrane Database of Systematic Reviews
Drug treatment for spinal muscular atrophy type I.2019The Cochrane Database of Systematic Reviews

Clinical Practice Guidelines