|Drug class||Antisense oligonucleotide|
|Main uses||Amyotrophic lateral sclerosis (ALS)|
|Side effects||Tiredness, pain, myelitis, increased intracranial pressure, aseptic meningitis|
|Chemical and physical data|
|Molar mass||7127.85 g·mol−1|
Tofersen, sold under the brand name Qalsody, is a medication used to treat amyotrophic lateral sclerosis (ALS). Specifically it is used in people with a mutation of the gene superoxide dismutase 1 (SOD1), which is present in 2% of cases. It is given by injection into the spinal cord.
Common side effects include tiredness, joint pain, increased CSF white blood cells, and muscle pain. Other side effect may include myelitis, increased intracranial pressure, and aseptic meningitis. It is an antisense oligonucleotide which decreases superoxide dismutase 1 production.
Tofersen was approved for medical use in the United States in 2023. As of 2023 it costs about 158,000 USD per year in the United States. It received orphan medication status in Europe in 2016.
Tofersen is indicated to treat people with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). This mutation is present in about 2% of cases.
Approval was based on a decrease in plasma neurofilament light chain rather than improved outcomes for the people in question.
It is given as a dose of 100 mg every two weeks for 3 doses and than every 4 weeks after that.
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. 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). The participants were approximately 43% female; 57% male; 64% White; and 8% Asian. The average age was 49.8 years (range from 23 to 78 years).
Society and culture
Only around 1-2% of ALS cases diagnosed in the United States each year carry the specific SOD1 mutation targeted by the drug. Fewer than 500 people a year are expected to be eligible for the drug, which is expected to cost over $100,000 for a year's treatment.
- "Qalsody- tofersen injection". DailyMed. 25 April 2023. Archived from the original on 8 May 2023. Retrieved 10 June 2023.
- "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.
- Robbins, Rebecca (25 April 2023). "F.D.A. Approves Drug for Rare Form of A.L.S." The New York Times. Archived from the original on 25 April 2023. Retrieved 22 June 2023.
- "EU/3/16/1732". European Medicines Agency. 17 September 2018. Archived from the original on 3 May 2023. Retrieved 22 June 2023.
- Liu A (1 May 2019). "Biogen's antisense ALS drug shows promise in early clinical trial". FierceBiotech. Archived from the original on 2 February 2023. Retrieved 25 April 2023.
- Langreth R (22 March 2023). "Biogen's ALS Drug Gets Partial Backing From FDA Panel". Bloomberg News. Retrieved 25 April 2023.
- Berdyński M, Miszta P, Safranow K, Andersen PM, Morita M, Filipek S, et al. (January 2022). "SOD1 mutations associated with amyotrophic lateral sclerosis analysis of variant severity". Scientific Reports. 12 (1): 103. Bibcode:2022NatSR..12..103B. doi:10.1038/s41598-021-03891-8. PMC 8742055. PMID 34996976.
- Constantino A (25 April 2023). "FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease". CNBC. Archived from the original on 25 April 2023. Retrieved 25 April 2023.
- Constantino A (22 March 2023). "FDA advisors vote against effectiveness of Biogen's ALS drug for rare and aggressive form of the disease". CNBC. Archived from the original on 10 April 2023. Retrieved 25 April 2023.
- Robins R (25 April 2023). "F.D.A. Approves Drug for Rare Form of A.L.S." The New York Times. Archived from the original on 25 April 2023. Retrieved 25 April 2023.