Nusinersen

Chemical formula: C₂₃₄H₃₄₀N₆₁O₁₂₈P₁₇S  Molecular mass: 7,127 g/mol 

Active ingredient description

Nusinersen is an antisense oligonucleotide (ASO) which increases the proportion of exon 7 inclusion in survival motor neuron 2 (SMN2) messenger ribonucleic acid (mRNA) transcripts by binding to an intronic splice silencing site (ISS-N1) found in intron 7 of the SMN2 pre-messenger ribonucleic acid (pre-mRNA) and hence when SMN2 mRNA is produced, it can be translated into the functional full length SMN protein. SMA is a progressive neuromuscular disease resulting from mutations in chromosome 5q in the SMN1 gene. Gene SMN2, located near SMN1, is responsible for a small amount of SMN protein production.

Medicine classification

This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:

ATC code Group title Classification
M09AX07 M Musculo-skeletal system → M09 Other drugs for disorders of the musculo-skeletal system → M09A Other drugs for disorders of the musculo-skeletal system → M09AX Other drugs for disorders of the musculo-skeletal system
Discover more medicines within M09AX07

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Title Information Source Document Type  
SPINRAZA Solution for injection European Medicines Agency (EU) MPI, EU: SmPC

External identifiers

CAS Substance: 1258984-36-9
DrugBank Drug: DB13161
RxNorm Ingredient: 1863556
SNOMED-CT Concept: 723985005
Nusinersen (substance)
UNII Identifier: 5Z9SP3X666
NUSINERSEN

© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.