Nusinersen

What is Nusinersen?

Nusinersen (SPINRAZA) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide used in treating spinal muscular atrophy.

Nusinersen sodium

What are the uses of this medicine?

• This medicine is used for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Nusinersen sodium colored

How does this medicine work?

• 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.

Who Should Not Use this medicine ?

• This medicine have no usage limitation.

What drug interactions can this medicine cause?

• No clinically important drug interactions have been observed with SPINRAZA.

Is this medicine FDA approved?

• It was approved for use in the United States in 2016.

How should this medicine be used?

Conduct the following laboratory tests at baseline and prior to each dose of SPINRAZA and as clinically needed:

• Platelet count
• Prothrombin time; activated partial thromboplastin time
• Quantitative spot urine protein testing

Recommended Dosage:

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

Administration

• SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures.
• Store SPINRAZA in the carton in a refrigerator until time of use.
• Allow the SPINRAZA vial to warm to room temperature (25° C/77° F) prior to administration.
• Do not use external heat sources.
• Inspect the SPINRAZA vial for particulate matter and discoloration prior to administration.
• Do not administer SPINRAZA if visible particulates are observed or if the liquid in the vial is discolored.
• The use of external filters is not required.
• Withdraw 12 mg (5 mL) of SPINRAZA from the single-dose vial into a syringe and discard unused contents of the vial.
• Administer SPINRAZA within 4 hours of removal from vial.
• Consider sedation as indicated by the clinical condition of the patient.
• Consider ultrasound or other imaging techniques to guide intrathecal administration of SPINRAZA, particularly in younger patients.
• Prior to administration, remove 5 mL of cerebrospinal fluid.
• Administer SPINRAZA as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle.
• Do not administer SPINRAZA in areas of the skin where there are signs of infection or inflammation

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

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

This medicine is available in fallowing brand namesː

• SPINRAZA

What side effects can this medication cause?

The most common side effects of this medicine include:

• lower respiratory infection and constipation in patients with infantile-onset SMA
• pyrexia, headache, vomiting, and back pain in patients with later-onset SMA

What special precautions should I follow?

• Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Increased risk for bleeding complications; testing required at baseline and before each dose and as clinically needed.
• Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Quantitative spot urine protein testing required at baseline and prior to each dose.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no adequate data on the developmental risk associated with the use of SPINRAZA in pregnant women.

Can this medicine be used in children?

• The safety and effectiveness of SPINRAZA in pediatric patients from newborn to 17 years have been established.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• Nusinersen

Inactive ingredients:

• Sodium phosphate, monobasic, dihydrate
• Sodium phosphate, dibasic, anhydrous
• Sodium Chloride
• Potassium Chloride
• Calcium chloride
• Magnesium chloride
• Water

Who manufactures and distributes this medicine?

• Packager: Biogen

What should I know about storage and disposal of this medication?

• Store in a refrigerator between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light.
• Do not freeze.
• SPINRAZA should be protected from light and kept in the original carton until time of use.
• If no refrigeration is available, SPINRAZA may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days.
• Prior to administration, unopened vials of SPINRAZA 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).

• Dailymed label info on Nusinersen
• FDA Nusinersen

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