nusinersen sodium: clinical use

Prescribing considerations

• Initiate only under a physician experienced in managing spinal muscular atrophy, following individualised assessment of expected benefit and risk.
• Profound hypotonia with respiratory failure present at birth was not studied and may limit clinically meaningful benefit.
• Administration requires lumbar-puncture expertise; consider sedation and imaging guidance where clinically appropriate.
• Do not inject through skin showing infection or inflammation.
• Evidence in renal or hepatic impairment is limited.

Contraindications and cautions

• Hypersensitivity to nusinersen or any excipient.
• Carefully reassess continued treatment if a ventriculoperitoneal shunt is required because benefits and risks in this setting are unknown.

Monitoring

• Review clinical presentation, response and ongoing benefit-risk regularly.
• Consider platelet count and coagulation testing before administration when clinically indicated.
• Consider first-morning urine protein testing when clinically indicated; investigate persistent proteinuria.
• Monitor for lumbar-puncture complications, hypersensitivity, meningitis, arachnoiditis and communicating hydrocephalus.

Clinical pharmacology

Nusinersen is an antisense oligonucleotide that binds the ISS-N1 site in SMN2 pre-mRNA, promoting exon 7 retention and production of full-length SMN protein. Following intrathecal administration it distributes through central nervous system tissues, undergoes slow exonuclease-mediated hydrolysis and is expected to be eliminated mainly in urine.

Formulation and product differences

• The selected presentation is a clear, colourless solution in a single-use vial for intrathecal injection.
• It must not be diluted and is considered essentially sodium-free and potassium-free.
• The vial requires refrigerated, light-protected storage and must not be frozen.

Injection

Route: Parenteral

Strengths: 12 mg, 28 mg, 50 mg