Drug reference
Nusinersen is an antisense oligonucleotide that increases the production of survival motor neurone (SMN) protein, thereby helping to compensate for the defect in the SMN1 gene found in 5q spinal muscular atrophy. They prolong the action of acetylcholine by inhibiting the action of the enzyme acetylcholinesterase.
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5q spinal muscular atrophy (initiated by a specialist) ▶BY INTRATHECAL INJECTION ▶Adult: Initially 12 mg for 4 doses, on days 0, 14, 28 and 63, then 12 mg every 4 months, for advice on missed doses—consult product literature Musculoskeletal system
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Risk factors for renal toxicity—monitor urine protein (preferably using a first morning urine specimen). risk factors for thrombocytopenia and coagulation disorders—monitor platelet and coagulation profile before treatment Arrhythmias. asthma (extreme caution). atropine or other antidote to muscarinic effects may be necessary (particularly when neostigmine is given by injection) but not given routinely because it may mask signs of overdosage. bradycardia. epilepsy. hyperthyroidism. hypotension. parkinsonism. peptic ulceration. recent myocardial infarction. vagotonia CAUTIONS, FURTHER INFORMATION ▶Elderly Prescription potentially inappropriate (STOPP criteria) in patients with a known history of persistent bradycardia (heart rate less than 60 beats per minute), heart block, or recurrent unexplained syncope, or concurrent treatment with drugs that reduce heart rate (risk of cardiac conduction failure, syncope and injury). See also Prescribing in the elderly p. 33.
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NICE decisions ▶Nusinersen for treating spinal muscular atrophy (July 2019) NICE TA588 Nusinersen (Spinraza ®) is recommended as an option for treating 5q spinal muscular atrophy (SMA) only if: . patients have pre-symptomatic SMA, or SMA types 1, 2 or 3, and . the conditions in the managed access agreement are followed. www.nice.org.uk/guidance/ta588
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Intestinal obstruction. urinary obstruction Musculoskeletal system
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MHRA/CHM ADVICE: NUSINERSEN (SPINRAZA ®): REPORTS OF COMMUNICATING HYDROCEPHALUS NOT RELATED TO MENINGITIS OR BLEEDING (JULY 2018) Communicating hydrocephalus not related to meningitis or bleeding has been reported in patients treated with Spinraza ®. Patients and caregivers should be informed about the signs and symptoms of hydrocephalus before Spinraza ® is started and should be instructed to seek medical attention in case of: persistent vomiting or headache, unexplained decrease in consciousness, and in children increase in head circumference. Patients with signs and symptoms suggestive of hydrocephalus should be further investigated by a physician with expertise in its management.
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Manufacturer advises avoid—no information available. Manufacturer advises use only if potential benefit outweighs risk.
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Manufacturer advises avoid—no information available. Amount probably too small to be harmful. eiiii F above
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Manufacturer advises close monitoring—safety and efficacy not established. l HANDLING AND STORAGE Manufacturer advises store in a refrigerator (2–8 °C); may be stored (in the original carton, protected from light) at or below 30 °C, for up to 14 days.
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Abdominal cramps. diarrhoea. excessive tearing. hypersalivation. nausea. vomiting Overdose Signs of overdosage include bronchoconstriction, increased bronchial secretions, lacrimation, excessive sweating, involuntary defaecation, involuntary micturition, miosis, nystagmus, bradycardia, heart block, arrhythmias, hypotension, agitation, excessive dreaming, and weakness eventually leading to fasciculation and paralysis.
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There can be variation in the licensing of different medicines containing the same drug. Solution for injection ▶Spinraza (Biogen Idec Ltd) A Nusinersen (as Nusinersen sodium) 2.4 mg per 1 ml Spinraza 12mg/5ml solution for injection vials | 1 vial P £75,000.00 3.2 Myasthenia gravis and Lambert-Eaton myasthenic syndrome ANTICHOLINESTERASES Anticholinesterases f
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Initially 12 mg for 4 doses, on days 0, 14, 28 and
First-line fragment. Read the complete Indications and Dose section above.
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