Tofersen
Medication / From Wikipedia, the free encyclopedia
Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS).[2] 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.[2]
Quick Facts Clinical data, Trade names ...
Clinical data | |
---|---|
Trade names | Qalsody |
AHFS/Drugs.com | Monograph |
MedlinePlus | a623024 |
License data | |
Routes of administration | Intrathecal |
ATC code | |
Legal status | |
Legal status | |
Identifiers | |
CAS Number | |
DrugBank | |
UNII | |
KEGG | |
Chemical and physical data | |
Formula | C230H317N72O123P19S15 |
Molar mass | 7127.85 g·mol−1 |
Close
The most common side effects include fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).[2]
Tofersen was approved for medical use in the United States in April 2023,[2][4] and in the European Union in February 2024.[3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]