 | |
| 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 |
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 amyotrophic lateral sclerosis. It is administered as an intrathecal injection.[2]
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][5] and in the European Union in May 2024.[3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[6]
- ^ "Qalsody- tofersen injection". DailyMed. 25 April 2023. Archived from the original on 8 May 2023. Retrieved 10 June 2023.
- ^ a b c d e "FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene" (Press release). U.S. Food and Drug Administration (FDA). 25 April 2023. Archived from the original on 25 April 2023. Retrieved 25 April 2023.
This article incorporates text from this source, which is in the public domain.
- ^ a b Cite error: The named reference
Qalsody EPARwas invoked but never defined (see the help page). - ^ Cite error: The named reference
Qalsody PIwas invoked but never defined (see the help page). - ^ "FDA Grants Accelerated Approval for Qalsody (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS" (Press release). Biogen. 25 April 2023. Archived from the original on 25 April 2023. Retrieved 25 April 2023 – via GlobeNewswire.
- ^ New Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024.
