Clinical data | |
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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 ALS. 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][3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[4]
Tofersen is indicated 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 by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen.[5][6]
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 US Food and Drug Administration (FDA) granted the application for tofersen priority review, orphan drug, and fast track designations.[2][4]
Only around 1-2% of ALS cases diagnosed in the United States each year carry the specific SOD1 mutation targeted by the drug.[7] 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][8][9][10]
In February 2024, the Committee for Medicinal Products for Human Use of the European 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.[11][12] The applicant for this medicinal product is Biogen Netherlands B.V.[11]