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Generic name: nusinersen (injection)
Brand name: Spinraza
Dosage form: intrathecal solution (12 mg/5 mL)
Drug class: Miscellaneous uncategorized agents

Medically reviewed by  A Ras MD.

What is nusinersen used for?

Nusinersen is  prescription medicine that is used to treat spinal muscular atrophy.


Nusinersen is a modified antisense oligonucleotide, where the 2′-hydroxy groups of the ribofuranosyl rings are replaced with 2′-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. The structural formula is:


SPINRAZA is supplied as a sterile, preservative-free, colorless solution for intrathecal use in a single-dose glass vial. Each 1 mL solution contains 2.4 mg of nusinersen (equivalent to 2.53 mg of nusinersen sodium salt). Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP, magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium chloride (8.77 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate monobasic dihydrate (0.05 mg) USP, and Water for Injection USP. The product may contain hydrochloric acid or sodium hydroxide to adjust pH. The pH is ~7.2.

The molecular formula of SPINRAZA is C234H323N61O128P17S17Na17 and the molecular weight is 7501.0 daltons.

Mechanism of Action

SPINRAZA is 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, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

Before taking nusinersen, tell your doctor:

  • If you are allergic to nusinersen; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take nusinersen with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take nusinersen?

  • Tell all of your health care providers that you take nusinersen. This includes your doctors, nurses, pharmacists, and dentists.
  • Have blood work checked as you have been told by the doctor. Talk with the doctor.
  • Have your urine checked as you have been told by your doctor.
  • You may bleed more easily. Be careful and avoid injury. Use a soft toothbrush and an electric razor.
  • Kidney problems have happened with drugs like this one. Sometimes, these have been deadly. Talk with the doctor.
  • Tell your doctor if you are pregnant, plan on getting pregnant, or are breast-feeding. You will need to talk about the benefits and risks to you and the baby.

How is nusinersen best taken?

Use nusinersen as ordered by your doctor. Read all information given to you. Follow all instructions closely.

  • It is given as a shot into the spine.

What do I do if I miss a dose?

  • Call your doctor to find out what to do.

What are the side effects of nusinersen that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

  • Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
  • Signs of bleeding like throwing up or coughing up blood; vomit that looks like coffee grounds; blood in the urine; black, red, or tarry stools; bleeding from the gums; abnormal vaginal bleeding; bruises without a cause or that get bigger; or bleeding you cannot stop.
  • Signs of kidney problems like unable to pass urine, change in how much urine is passed, blood in the urine, or a big weight gain.
  • Signs of a urinary tract infection (UTI) like blood in the urine, burning or pain when passing urine, feeling the need to pass urine often or right away, fever, lower stomach pain, or pelvic pain.
  • Chills.
  • Change in color of sputum.
  • Shortness of breath.
  • Cough.
  • Ear pain.
  • Falls.

What are some other side effects of nusinersen?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

  • Signs of a common cold.
  • Constipation.
  • Headache.
  • Back pain.
  • Gas.
  • Weight loss.
  • Throwing up.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out nusinersen?

  • If you need to store nusinersen at home, talk with your doctor, nurse, or pharmacist about how to store it.


Principal Display Panel – Spinraza 12mg/5ml Carton Label

  • NDC 64406-058-01
  • Spinraza™
  • (nusinersen)
  • Injection
  • 12 mg/5 mL
  • (2.4 mg/mL)
  • Sterile solution for
    Intrathecal Injection Only
  • Rx Only
  • Single Use Vial
  • Discard Unused Portion
Principal Display Panel - Spinraza 12mg/5ml Carton Label



Principal Display Panel – Spinraza 12mg/5ml Vial Label

  • NDC 64406-058-01
  • Spinraza™
  • (nusinersen) injection
  • 12 mg/5 mL (2.4 mg/mL)
  • For Intrathecal Injection Only
Principal Display Panel - Spinraza 12mg/5ml Vial Label


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