Tofersen

Tofersen
Clinical data
Trade namesQalsody
AHFS/Drugs.comMonograph
MedlinePlusa623024
License data
Routes of
administration		Intrathecal
ATC code
Legal status
Legal status
Identifiers
  • all-P-ambo-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'→5')-2'-O-(2-methoxyethyl)-guanylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridine
CAS Number
PubChem CID
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC230H317N72O123P19S15
Molar mass7127.85 g·mol−1
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  • Key:AZBXTZOFHIRMCD-SIVATHJTSA-N

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with ALS. It is administered as an intrathecal injection.

Tofersen is approved for medical use in the US and EU. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.

Common side effects include fatigue, arthralgia, increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia.

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