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Nusinersen Sodium

Class: Other Miscellaneous Therapeutic Agents
Brands: Spinraza


Nusinersen sodium is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide.1

Uses for Nusinersen Sodium

Nusinersen sodium has the following uses:

Nusinersen sodium is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.1

Nusinersen Sodium Dosage and Administration


Nusinersen sodium is available in the following dosage form(s) and strength(s):

Injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial.1


It is essential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

Nusinersen sodium is administered intrathecally.1

    Dosing Information1
  • Available as nusinersen sodium; dosage expressed in terms of nusinersen.1

  • The recommended dosage is 12 mg (5 mL) per administration.1

  • Initiate nusinersen sodium treatment with 4 loading doses; the first three loading doses should be administered at 14-day intervals; the 4th loading dose should be administered 30 days after the 3rd dose; a maintenance dose should be administered once every 4 months thereafter.1

    Important Preparation and Administration Instructions1
  • Allow to warm to room temperature prior to administration. 1

  • Administer within 4 hours of removal from vial. 1

  • Prior to administration, remove 5 mL of cerebrospinal fluid. 1

  • Administer as intrathecal bolus injection over 1 to 3 minutes. 1

    Laboratory Testing and Monitoring to Assess Safety1
  • At baseline and prior to each dose, obtain a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing.1

Cautions for Nusinersen Sodium


None. 1


Thrombocytopenia and Coagulation Abnormalities

Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides.1

In a clinical study, 6 of 56 (11%) nusinersen-treated patients with normal or above normal platelet levels at baseline developed a platelet level below the lower limit of normal, compared to 0 of 28 sham-procedure control patients. No patient had a platelet count less than 50,000 cells per microliter in this study and no patient developed a sustained low platelet count despite continued drug exposure.1

Because of the risk of thrombocytopenia and coagulation abnormalities from nusinersen, patients may be at increased risk of bleeding complications.1

Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of nusinersen sodium and as clinically needed.1

Renal Toxicity

Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides.1

Nusinersen is present in and excreted by the kidney. In a clinical study (mean treatment exposure 7 months), 17 of 51 (33%) nusinersen-treated patients had elevated urine protein, compared to 5 of 25 (20%) sham-control patients. In a group of later-onset spinal muscular atrophy patients (mean treatment exposure 34 months), 36 of 52 (69%) had elevated urine protein. No elevations in serum creatinine or cystatin C were observed in these studies. Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of nusinersen. For urinary protein concentration greater than 0.2 g/L, consider repeat testing and further evaluation. 1

Specific Populations


There are no adequate data on the developmental risk associated with the use of nusinersen sodium in pregnant women. No adverse effects on embryofetal development were observed in animal studies in which nusinersen sodium was administered by subcutaneous injection to mice and rabbits during pregnancy.1

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. The background risk of major birth defects and miscarriage for the indicated population is unknown.1

When nusinersen sodium (0, 3, 10, or 25 mg/kg) was administered subcutaneously to male and female mice every other day prior to and during mating and continuing in females throughout organogenesis, no adverse effects on embryofetal development were observed. Subcutaneous administration of nusinersen sodium (0, 6, 12.6, or 25 mg/kg) to pregnant rabbits every other day throughout organogenesis produced no evidence of embryofetal developmental toxicity. 1


There are no data on the presence of nusinersen sodium in human milk, the effects on the breastfed infant, or the effects of the drug on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for nusinersen sodium and any potential adverse effects on the breastfed infant from nusinersen sodium or from the underlying maternal condition.1

Pediatric Use

The safety and effectiveness of nusinersen sodium in pediatric patients from newborn to 17 years have been established.1

In intrathecal toxicity studies in juvenile monkeys, administration of nusinersen sodium (0, 0.3, 1, or 3 mg/dose for 14 weeks and 0, 0.3, 1, or 4 mg/dose for 53 weeks) resulted in brain histopathology (neuronal vacuolation and necrosis/cellular debris in the hippocampus) at the mid and high doses and acute, transient deficits in lower spinal reflexes at the high dose in each study. In addition, possible neurobehavioral deficits were observed on a learning and memory test at the high dose in the 53-week monkey study. The no-effect dose for neurohistopathology in monkeys (0.3 mg/dose) is approximately equivalent to the human dose when calculated on a yearly basis and corrected for the species difference in CSF volume.1

Geriatric Use

Spinal muscular atrophy is largely a disease of children and young adults; therefore, there is no geriatric experience with nusinersen.1

Common Adverse Effects

The most common adverse reactions that occurred in at least 20% of nusinersen-treated patients and occurred at least 5% more frequently than in control patients were lower respiratory infection, upper respiratory infection, and constipation.1

Drug Interactions

Specific Drugs

It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

Please see product labeling for drug interaction information.


Mechanism Of Action

Nusinersen sodium is an antisense oligonucleotide designed to treat spinal muscular atrophy caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of spinal muscular atrophy, nusinersen sodium was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.1

Advice to Patients

Thrombocytopenia and Coagulation Abnormalities

Inform patients and caregivers that nusinersen sodium could increase the risk of bleeding. Inform patients and caregivers of the importance of obtaining blood laboratory testing at baseline and prior to each dose to monitor for signs of increased potential for bleeding. Instruct patients and caregivers to seek medical attention if unexpected bleeding occurs.1

Renal Toxicity

Inform patients and caregivers that nusinersen sodium could cause renal toxicity. Inform patients and caregivers of the importance of obtaining urine testing at baseline and prior to each dose to monitor for signs of potential renal toxicity.1

Additional Information

AHFS First Release. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.


Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Nusinersen Sodium


Dosage Forms


Brand Names



Injection, Solution

2.4 mg /1 mL (of nusinersen)



AHFS Drug Information. © Copyright 2017, Selected Revisions January 9, 2017. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.


1. Biogen. Spinraza (Nusinersen) INTRATHECAL prescribing information. 2016 Dec.