Nusinersen

Class: Antisense Oligonucleotides
Chemical Name: [2′-O-(2-Methoxyethyl)](P-thio)(m5U-m5C-A-m5C-m5U-m5U-m5U-m5C-A-m5U-A-A-m5U-G-m5C-m5U-G-G) RNA
Molecular Formula: C₂₃₄H₃₂₃N₆₁O₁₂₈P₁₇S₁₇Na₁₇
CAS Number: 1258984-36-9
Brands: Spinraza

Medically reviewed by Drugs.com on Jan 19, 2022. Written by ASHP.

Introduction

Antisense oligonucleotide that binds to the pre-mRNA of survival motor neuron 2 (SMN2).

Uses for Nusinersen

Spinal Muscular Atrophy

Management of spinal muscular atrophy (designated an orphan drug by FDA for use in this condition).

Associated with substantial improvement in motor milestones (e.g., ability to sit unassisted, stand, walk).

May be used across full range of patients with spinal muscular atrophy (e.g., infantile-onset to later-onset disease). Available evidence to date supports early initiation of therapy.

Nusinersen Dosage and Administration

General

• Monitor platelet counts, prothrombin time (PT), activated partial thromboplastin time (aPTT), and urine protein levels at baseline, prior to each dose, and as clinically indicated. (See Cautions.)

• Consider use of sedation during administration as indicated by patient's clinical condition.

• Consider use of ultrasound or other imaging techniques to guide intrathecal administration, especially in younger patients.

Administration

Intrathecal Administration

Administer by intrathecal injection only. Administer by, or under the direction of, a clinician experienced in performing lumbar punctures. For specific procedures and techniques of administration, consult manufacturer's full prescribing information.

Prior to administration, allow vials to warm to room temperature; do not use external heat sources for warming. Solution should be clear and colorless; do not use if visible particulates or discoloration observed.

Prior to administration, remove 5 mL of CSF. To prepare dose for intrathecal injection, withdraw 5 mL (12 mg) of nusinersen injection solution into a syringe using proper aseptic technique; administer by direct (“bolus”) intrathecal injection over 1–3 minutes using a spinal anesthesia needle. Do not inject into areas of skin showing signs of infection or inflammation.

Administer drug within 4 hours after removal from vial. Vials contain no preservatives and are intended for single use only; discard unused contents.

If a loading dose is delayed or missed, administer delayed or missed dose as soon as possible with at least 14 days between doses; resume regular dosing schedule thereafter. If a maintenance dose is delayed or missed, administer delayed or missed dose as soon as possible and resume regular dosing schedule.

Dosage

Dosage of nusinersen sodium expressed in terms of nusinersen.

Pediatric Patients

Spinal Muscular Atrophy

Intrathecal

Initiate therapy with a series of 4 loading doses of 12 mg (5 mL) each as follows: administer first 3 doses every 14 days and the fourth dose 30 d...