Nusinersen

Nusinersen is used for spinal muscular atrophy (SMA) in children and adults.

Nusinersen is available under the following different brand names: Spinraza.

Dosages of Nusinersen:

Dosage Forms and Strengths

Solution for Intrathecal Injection

• 12mg/5mL single-dose vial

Dosage Considerations – Should be Given as Follows:

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

Laboratory tests

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

Common side effects of Nusinersen include:

• Headache
• Lower respiratory infection
• Upper respiratory infection
• Upper respiratory tract congestion
• Back pain
• Postlumbar puncture syndrome
• Elevated urine protein
• Constipation
• Partial or complete lung collapse
• Low blood platelets (thrombocytopenia)
• Aspiration
• Ear infection
• Emergent treatment anti-drug antibodies

Postmarketing side effects of nusinersen reported include:

• Hydrocephalus
• Aseptic meningitis
• Hypersensitivity reactions (e.g. angioedema, hives, rash)

This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.

• Nusinersen has no listed severe interactions with other drugs.
• Nusinersen has no listed serious interactions with other drugs.
• Nusinersen has no listed moderate interactions with other drugs.
• Nusinersen has no listed mild interactions with other drugs.

This document does not contain all possible interactions. Therefore, before using this product, tell your doctor or pharmacist of all the products you use. Keep a list of all your medications with you, and share the list with your doctor and pharmacist. Check with your physician if you have health questions or concerns.

Warnings

• This medication contains nusinersen. Do not take Spinraza if you are allergic to nusinersen or any ingredients contained in this drug.

Contraindications

• None

Effects of Drug Abuse

• No information is available.

Short-Term Effects

• See "What Are Side Effects Associated with Using Nusinersen?"

Long-Term Effects

• See "What Are Side Effects Associated with Using Nusinersen?"

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 before 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 before each dose
• For urinary protein concentration greater than 0.2 g/L, consider repeat testing and further evaluation

Re-elevation of coagulation parameters

• In a limited number of patients in the clinical program, between 12-24 hours after administrating nusinersen 5 g, elevated coagulation parameters (e.g., aPTT, ECT) have been observed
• If reappearance of clinically relevant bleeding together with elevated coagulation parameters is observed after administering nusinersen 5 g, administration of an additional 5 g dose may be considered
• Similarly, patients who require a second emergency surgery/urgent procedure and have elevated coagulation parameters may receive an additional 5-g dose
• The safety and effectiveness of repeat treatment with nusinersen have not been established

Pregnancy and Lactation

• There are no adequate data on the developmental risk associated with the use of nusinersen 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. Consult your doctor.
• It is unknown if nusinersen is distributed in human breast milk. Nusinersen is detected in the 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 nusinersen, and any potential adverse effects on the breastfed infant from the drug or the underlying maternal condition.