nusinersen (Rx)

Brand and Other Names:Spinraza
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Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

solution for intrathecal injection

  • 12mg/5mL single-dose vial

Spinal Muscular Atrophy

Indicated for spinal muscular atrophy (SMA) in children and adults

12 mg intrathecally per administration

Initial: 4 loading doses; administer the first 3 doses at 14-day intervals and the fourth dose 30 days after the third dose

Maintenance: One dose every 4 months

Also see Administration

Dosing Considerations

Laboratory tests

  • Conduct the following laboratory tests at baseline and prior to each dose and as clinically needed
  • Platelet count
  • Prothrombin time (PT); activated partial thromboplastin time (aPTT)
  • Quantitative spot urine protein testing

Dosage Forms & Strengths

solution for intrathecal injection

  • 12mg/5mL single-dose vial

Spinal Muscular Atrophy

Indicated for spinal muscular atrophy (SMA) in children and adults

Safety and effectiveness have been established in children aged newborn to 17 years

12 mg intrathecally per administration

Initial: 4 loading doses; administer the first 3 doses at 14-day intervals and the fourth dose 30 days after the third dose

Maintenance: One dose every 4 months

Also see Administration

Dosing Considerations

Laboratory tests

  • Conduct the following laboratory tests at baseline and prior to each dose and as clinically needed
  • Platelet count
  • Prothrombin time (PT); activated partial thromboplastin time (aPTT)
  • Quantitative spot urine protein testing
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Adverse Effects

Data are from controlled study in infants

>10%

Headache (50%)

Lower respiratory infection (43%)

Back pain (41%)

Postlumbar puncture syndrome (41%)

Upper respiratory infection (39%)

Elevated urine protein (33%)

Constipation (30%)

Atelectasis (14%)

Thrombocytopenia (11%)

1-10%

Upper respiratory tract congestion (6%)

Aspiration (5%)

Ear infection (5%)

Emergent treatment antidrug antibodies (4%)

Postmarketing Reports

Hydrocephalus

Aseptic meningitis

Hypersensitivity reactions (e.g. angioedema, urticaria, rash)

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Warnings

Contraindications

None

Cautions

Coagulation

  • Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides
  • Obtain platelet count and coagulation laboratory testing at baseline and prior to each administration and as clinically needed

Renal toxicity

  • Nusinersen is present in and excreted by the kidney
  • Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides
  • Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose
  • For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation
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Pregnancy

Pregnancy

There are no adequate data on the developmental risk associated with use in pregnant women

No adverse effects on embryofetal development were observed in animal studies in which nusinersen was administered by subcutaneous injection to mice and rabbits during pregnancy

Lactation

Unknown if distributed in human breast milk; detected in milk of lactating mice when administered by subcutaneous injection

Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for the drug, and any potential adverse effects on the breastfed infant from the drug or from the underlying maternal condition

Pregnancy Categories

A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA: Information not available.

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Pharmacology

Mechanism of Action

An antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency

Using in vitro assays and studies in transgenic animal models of SMA, nusinersen was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein

Absorption

Trough plasma levels relatively low, compared to trough CSF concentration

Peak plasma time: 1.7-6 hr

Distribution

CSF and peripheral tissues (eg, skeletal muscle, liver, kidney)

Metabolism

Metabolized via exonuclease (3’- and 5’)-mediated hydrolysis

Not a substrate for, or inhibitor, or inducer of CYP450 enzymes

Elimination

Half-life: 135-177 days (CSF); 63-87 days (plasma)

Excretion: primary route of elimination is likely by urinary excretion; at 24 hr, only 0.5% of the administered dose was recovered in the urine

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Administration

Intrathecal Preparation

Obtain laboratory testing before each dose (see Dosing)

Use aseptic technique

Each vial is intended for single dose only

Store drug in the carton in a refrigerator until time of use

Allow the vial to warm to room temperature (25°C/77°F) prior to administration; do not use external heat sources

Inspect vial content for particulate matter and discoloration prior to administration; do not administer if visible particulates are observed or if the liquid in the vial is discolored (should be clear and colorless)

Use of external filters not required

Withdraw 12 mg (5 mL) from the single-dose vial into a syringe and discard unused contents (ie, overfill) of the vial

Administer within 4 hr of removal from vial

Intrathecal Administration

For intrathecal use only

Consider sedation as indicated by the clinical condition of the patient

Consider ultrasound or other imaging techniques to guide intrathecal administration of, particularly in younger patients

Prior to administration, remove 5 mL of cerebrospinal fluid

Administer as an intrathecal bolus injection over 1-3 minutes using a spinal anesthesia needle

Do not administer in areas of the skin where there are signs of infection or inflammation

Missed dose

  • If a loading dose is delayed or missed, administer as soon as possible, with at least 14-days between doses and continue dosing as prescribed
  • If a maintenance dose is delayed or missed, administer as soon as possible and continue dosing every 4 month

Storage

Store in a refrigerator between 2-8°C (36-46°F) in the original carton to protect from light

Do not freeze

Should be protected from light and kept in the original carton until time of use

If no refrigeration is available, may be stored in its original carton, protected from light at ≤30°C (86°F) for up to 14 days

Prior to administration, unopened vials can be removed from and returned to the refrigerator, if necessary

If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours at a temperature that does not exceed 25°C (77°F)

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Images

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Formulary

FormularyPatient Discounts

Adding plans allows you to compare formulary status to other drugs in the same class.

To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.

Adding plans allows you to:

  • View the formulary and any restrictions for each plan.
  • Manage and view all your plans together – even plans in different states.
  • Compare formulary status to other drugs in the same class.
  • Access your plan list on any device – mobile or desktop.

The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.