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Generic name: nusinersen systemic

Brand names: Spinraza

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intrathecal [preservative free]:

Spinraza: 12 mg/5 mL (5 mL)


Mechanism of Action

Treats spinal muscular atrophy caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency by binding to a specific sequence in the intron downstream of exon 7 of the SMN2 messenger ribonucleic acid (mRNA) transcript and increase production of full-length SMN protein.



Distributed within the CNS and peripheral tissues


Via exonuclease (3’- and 5’)-mediated hydrolysis



Time to Peak

Median range: 1.7 to 6 hours

Half-Life Elimination

Terminal (mean range): CSF: 135 to 177 days; Plasma: 63 to 87 days

Use: Labeled Indications

Spinal muscular atrophy: Treatment of spinal muscular atrophy (SMA)


There are no contraindications listed in the manufacturer's labeling.

Canadian labeling: Additional contraindications (not in US labeling): Known or suspected hypersensitivity to nusinersen or any component of the formulation.

Dosage and Administration

Dosing: Adult

Spinal muscular atrophy: Intrathecal: Loading dose: 12 mg once every 14 days for 3 doses; then 12 mg once 30 days after the third dose. Maintenance: 12 mg once every 4 months

Dosing: Pediatric

Spinal muscular atrophy: Infants, Children, and Adolescents:

Loading dose: Intrathecal: 12 mg once every 14 days for 3 doses; then 12 mg once 30 days after the third dose

Maintenance dose: Intrathecal: 12 mg once every 4 months


For intrathecal use only. Allow vial to warm to room temperature before use; do not use external heat sources. Prior to administration, remove 5 mL of cerebrospinal fluid. Administer as an intrathecal bolus injection over 1 to 3 minutes. Do not administer in areas with signs of infection or inflammation.


Store intact vials between 2°C to 8°C (36°F to 46°F) in original carton, protected from light. Do not freeze. May store intact vials at or below 30°C (86°F) for up to 14 days in original carton, protected from light. Intact vials in original carton may 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 not exceeding 25°C (77°F). Following removal from vial, administer solution within 4 hours; discard unused contents of the vial.

Drug Interactions

There are no known significant interactions.

Adverse Reactions


Central nervous system: Headache (29%)

Gastrointestinal: Constipation (35%), vomiting (29%), period of tooth development (18%)

Genitourinary: Proteinuria (58%)

Hematologic & oncologic: Thrombocytopenia (16%)

Neuromuscular & skeletal: Back pain (25%)

Respiratory: Lower respiratory tract infection (55%), atelectasis (18%)

Miscellaneous: Fever (43%)

1% to 10%:

Central nervous system: Fall (5%)

Endocrine & metabolic: Weight loss (5%)

Gastrointestinal: Flatulence (5%)

Genitourinary: Urinary tract infection (9%)

Hypersensitivity: Seasonal allergy (5%)

Immunologic: Antibody development (6%)

Otic: Otic infection (6%)

Respiratory: Respiratory congestion (5%; upper tract: 8%), epistaxis (7%)

<1%, postmarketing, and/or case reports: Angioedema, aseptic meningitis, hydrocephalus, hypersensitivity reaction, maculopapular rash, meningitis, serious infection, skin rash


Concerns related to adverse effects:

  • Hematologic effects: Coagulation abnormalities and thrombocytopenia (including acute severe thrombocytopenia), have been observed with some antisense oligonucleotides; increased risk of bleeding complications may occur. Perform a platelet count and coagulation testing at baseline, prior to each dose and as clinically needed.
  • Renal toxicity: Renal toxicity, including potentially fatal glomerulonephritis, has been observed with some antisense oligonucleotides. Conduct quantitative spot urine protein testing (preferably using first morning urine) at baseline and prior to each dose. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

Monitoring Parameters

Platelet count, coagulation tests (prothrombin time; activated partial thromboplastin time), and quantitative spot urine protein testing at baseline, prior to each dose, and as clinically indicated.


Pregnancy Considerations

Adverse events have been observed in some animal reproduction studies.

Patient Education

What is this drug used for?

  • It is used to treat spinal muscular atrophy.

Frequently reported side effects of this drug

  • Common cold symptoms
  • Constipation
  • Passing gas
  • Headache
  • Weight loss
  • Vomiting
  • Back pain

Other side effects of this drug: Talk with your doctor right away if you have any of these signs of:

  • Kidney problems like unable to pass urine, blood in the urine, change in amount of urine passed, or weight gain
  • Bleeding like vomiting blood or vomit that looks like coffee grounds; coughing up blood; blood in the urine; black, red, or tarry stools; bleeding from the gums; abnormal vaginal bleeding; bruises without a reason or that get bigger; or any severe or persistent bleeding
  • Urinary tract infection like blood in the urine, burning or painful urination, passing a lot of urine, fever, lower abdominal pain, or pelvic pain
  • Falls
  • Chills
  • Sputum discoloration
  • Shortness of breath
  • Cough
  • Ear pain
  • Signs of a significant reaction like wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about this medicine. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not specific medical advice and does not replace information you receive from the healthcare provider. You must talk with the healthcare provider for complete information about the risks and benefits of using this medicine.

Source: Wolters Kluwer Health. Last updated December 30, 2019.