Spinraza Nusinersen Sodium Injection

1. Indications and Usage
Spinal Muscular Atrophy (SMA):
Indicated for the treatment of patients with spinal muscular atrophy (SMA).
Applicability: Approved for infants, children, and adults across all SMA types.
2. Dosage and Administration
Route of Administration:
For Intrathecal Injection ONLY. Must be administered by a healthcare professional experienced in intrathecal injections.
Recommended Dosage:
Dose: 12 mg (5 mL) per dose.
Loading Dose: Administer at Day 0, Day 14, Day 28, and Day 63 (4th bolus).
Maintenance Dose: Administer once every 4 months after the loading dose.
Administration Instructions:
Pre-procedure: Drain cerebrospinal fluid (CSF) volume equivalent to the injected drug volume before administration.
Preparation: Allow the vial to reach room temperature naturally. Do not dilute or shake.
3. Mechanism of Action
Antisense Oligonucleotide (ASO):
Nusinersen is an ASO that selectively binds to a specific sequence in the SMN2 pre-mRNA transcript.
Splicing Modification:
It modulates splicing to increase the inclusion of Exon 7, thereby producing full-length, functional Survival of Motor Neuron (SMN) protein.
4. Safety and Warnings
Renal Toxicity:
Monitor for renal toxicity. Perform urinalysis (preferably first morning urine) and monitor protein levels before each dose.
Coagulation and Thrombocytopenia:
Monitor platelet counts and coagulation parameters (e.g., PT, aPTT) before each dose.
Post-Lumbar Puncture Complications:
Monitor for headache, back pain, vomiting, hypertension, tachycardia, and urinary retention.
Respiratory Infections:
Monitor for respiratory infections, as patients with SMA are at increased risk.
5. Adverse Reactions
Most Common:
Post-procedure complications (headache, back pain, vomiting), respiratory infections (pneumonia, influenza), constipation, and urinary retention.
6. Drug Interactions
Metabolism:
Nusinersen is not metabolized by CYP450 enzymes.
Anticoagulants:
Concomitant use with anticoagulants or antiplatelet agents may increase the risk of bleeding during lumbar puncture.
7. Pharmaceutical Information
Chemical Composition:
Active Ingredient: Nusinersen Sodium.
Available Strength: 12 mg/5 mL (2.4 mg/mL) in single-use vials.
Storage:
Store in a refrigerator at 2-8°C. Protect from light.