Tofersen

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 into the spinal cord.[2]

Tofersen
Clinical data
Trade namesQalsody
License data
Routes of
administration		Intrathecal
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC230H317N72O123P19S15
Molar mass7127.85 g·mol−1

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]

Medical uses

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]

History

Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen.[4][5]

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]

Society and culture

Economics

Only around 1-2% of ALS cases diagnosed in the United States each year carry the specific SOD1 mutation targeted by the drug.[6] 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][7][8][9]

References
  1. "Qalsody- tofersen injection". DailyMed. 25 April 2023. Archived from the original on 8 May 2023. Retrieved 10 June 2023.
  2. "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. Public Domain This article incorporates text from this source, which is in the public domain.
  3. "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.
  4. 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.
  5. Langreth R (22 March 2023). "Biogen's ALS Drug Gets Partial Backing From FDA Panel". Bloomberg News. Retrieved 25 April 2023.
  6. 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.
  7. 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.
  8. 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.
  9. 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.
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