 | |
| 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 nameQalsody, is amedication used for the treatment ofamyotrophic lateral sclerosis (ALS).[2] Tofersen is anantisense oligonucleotide that targets the production ofsuperoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. It is administered as anintrathecal 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 USFood and Drug Administration (FDA) considers it to be afirst-in-class medication.[6]
Tofersen isindicated to treat people with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).[2]
Tofersen was developed byIonis Pharmaceuticals and was licensed to, and co-developed by,Biogen.[7][8]
The effectiveness of tofersen was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 participants with weakness attributable to amyotrophic lateral sclerosis and a superoxide dismutase 1 (SOD-1) mutation confirmed by a central laboratory.[2] The study randomly assigned 108 participants in a 2:1 ratio to receive treatment with either tofersen 100 mg (n = 72) or placebo (n = 36) for 24 weeks (three loading doses followed by five maintenance doses).[2] The participants were approximately 43% female; 57% male; 64% White; and 8% Asian.[2] The average age was 49.8 years (range from 23 to 78 years).[2]
The stage IIIclinical trial was conducted by the Neuroscience Institute and Sheffield Institute for Translational Neuroscience (SITraN), both at the University of Sheffield.[9]
The USFood and Drug Administration (FDA) granted the application for tofersenpriority review,orphan drug, andfast track designations.[2][6]
Only around 1-2% of ALS cases diagnosed in the United States each year carry the specific SOD1 mutation targeted by the drug.[10] Fewer than 500 patients a year are expected to be eligible for the drug, which is expected to cost over $100,000 for a year's treatment.[2][11][12][13]
In February 2024, theCommittee for Medicinal Products for Human Use of theEuropean Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Qalsody, intended for the treatment of a type of amyotrophic lateral sclerosis caused by a defective superoxide dismutase 1 (SOD1) protein.[3][14] The applicant for this medicinal product is Biogen Netherlands B.V.[3] Tofersen was approved for medical use in the European Union in May 2024.[4]
This article incorporates text from this source, which is in thepublic domain.