Pharmacokinetics in children

Tmax (mean (range)): 1.7 - 6.0 h
T1/2 (mean): 135 - 177 days
[EPAR Spinraza]

dose recommendation of formulary compared to licensed use (on-label versus off-label)

No information is present at this moment.

Available formulations

No information is present at this moment.

Dosages

Renal impaiment in children > 3 months

No information available on dose adjustment in renal impairment.

The complete list of all undesirable drug reactions can be found in the national Summary of Product Characteristics (SmPC) – click here

Side effects in children

Very common: post-lumbar puncture syndrome (headache, vomiting, back pain)

Frequency unknown: meningitis, communicating hydrocephalus, aseptic meningitis and hypersensitivity (e.g. angioedema, urticaria and rash).

Formation (in approximately 4% of individuals) of antigenic drug antibodies (ADAs), without impact on side effects. With formation of ADAs, the response is persistent in some cases, and transient in others.

[SmPC Spinraza]

The complete list of all contra-indications can be found in the national Summary of Product Characteristics (SmPC) – click here

Contra-indications

No information available on specific contra indications in children.

The complete list of all warnings and precautions can be found in the national Summary of Product Characteristics (SmPC) – click here

Warnings & precautions in children

Laboratory tests (platelets, coagulation function) should be performed prior to administration, if clinically indicated, in view of the reporting of thrombocytopenia (including acute severe thrombocytopenia) and abnormalities of coagulation with other antisense oligonucleotides.

Administration: Depending on the clinical condition of the patient, sedation may be indicated around administration. Consider ultrasound or other imaging techniques, especially in younger patients or if scoliosis is present. There is a risk of side effects related to the puncture (headache, back pain, vomiting, infections such as meningitis).

Accumulation of cerebrospinal fluid: during the treatment, symptoms associated with (communicating) hydrocephalus may occur, such as persistent vomiting or headache, unexplained change in consciousness (listless, sleeping a lot, less alert). Have the patient report immediately when these symptoms occur and rule out increased pressure from cerebrospinal fluid and infection before diagnosing hydrocephalus. Consider the need for a ventriculoperitoneal shunt (VPS). The efficacy and safety of nusinersen in patients with a VPS have not been established; therefore, closely monitor such patients if a decision is made to continue treatment.

Nusinersen has not been studied in patients with significant hypotonia and respiratory failure at birth; these patients may not experience a clinically meaningful benefit due to a severe deficiency of the SMN protein.

Renal toxicity has been observed with the administration of other antisense oligonucleotides. On clinical indication, it is recommended to determine the protein content of the urine (preferably on a sample of the first morning urine). Consider further evaluation in case of persistently elevated urine protein levels.

Interactions

The complete list of all interactions can be found in the national Summary of Product Characteristics (SmPC) – click here

• MUSCULO-SKELETAL SYSTEM
• OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM

OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM

This pages provides a list of drugs from the same ATC class for comparison. This does not necessarily mean that these drugs are interchangeable.

Reference

1. Biogen Netherlands B.V., SmPC Spinraza 12 mg solution for injection (EU/1/17/1188/001) 02-02-2022, www.geneesmiddeleninformatiebank.nl

Changes

Therapeutic Drug Monitoring

Overdose