Chemical formula: C₂₃₄H₃₄₀N₆₁O₁₂₈P₁₇S Molecular mass: 7,127 g/mol
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.
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 | |
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 |
Nusinersen is an active ingredient of these brands:
United States (US)
Australia (AU)
Austria (AT)
Brazil (BR)
Croatia (HR)
Cyprus (CY)
Estonia (EE)
Finland (FI)
France (FR)
Hong Kong (HK)
Ireland (IE)
Israel (IL)
Italy (IT)
Japan (JP)
Lithuania (LT)
Netherlands (NL)
New Zealand (NZ)
Poland (PL)
Romania (RO)
Spain (ES)
Turkey (TR)
United Kingdom (UK)
Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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