Tofersen Explained

Width:300
Tradename:Qalsody
Dailymedid:Tofersen
Routes Of Administration:Intrathecal
Atc Prefix:N07
Atc Suffix:XX22
Legal Us:Rx-only
Legal Us Comment:[1] [2]
Legal Eu:Rx-only
Cas Number:2088232-70-4
Drugbank:DB14782
Unii:2NU6F9601K
Kegg:D11811
C:230
H:317
N:72
O:123
P:19
S:15

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). 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.

The most common side effects include fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).

Tofersen was approved for medical use in the United States in April 2023,[3] and in the European Union in February 2024. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[4]

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).

History

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. 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).

The stage III clinical trial was conducted by the Neuroscience Institute and Sheffield Institute for Translational Neuroscience (SITraN), both at the University of Sheffield.[7]

The US Food and Drug Administration (FDA) granted the application for tofersen priority review, orphan drug, and fast track designations.

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.[8] 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.[9] [10] [11]

Legal status

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.[12] The applicant for this medicinal product is Biogen Netherlands B.V.[13] The marketing authorisation in the EU was granted on 29 May 2024.[14]

Notes and References

  1. Web site: Qalsody- tofersen injection . DailyMed . 25 April 2023 . 10 June 2023 . 8 May 2023 . https://web.archive.org/web/20230508092955/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=81356b45-1cb7-4eef-88ea-e44cc18b47c5 . live .
  2. FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene . U.S. Food and Drug Administration (FDA) . 25 April 2023 . 25 April 2023 . 25 April 2023 . https://web.archive.org/web/20230425172813/https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene . live .
  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 . Biogen . GlobeNewswire . 25 April 2023 . 25 April 2023 . 25 April 2023 . https://web.archive.org/web/20230425191620/https://www.globenewswire.com/news-release/2023/04/25/2654404/0/en/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.html . live .
  4. New Drug Therapy Approvals 2023 . U.S. Food and Drug Administration (FDA) . January 2024 . PDF . 9 January 2024 . https://web.archive.org/web/20240110032419/https://www.fda.gov/media/175253/download . 10 January 2024 . live .
  5. News: Liu. Angus. 1 May 2019. Biogen's antisense ALS drug shows promise in early clinical trial. FierceBiotech. 25 April 2023. 2 February 2023. https://web.archive.org/web/20230202115409/https://www.fiercebiotech.com/research/biogen-s-antisense-als-drug-shows-promise-early-clinical-study. live .
  6. News: Langreth. Robert. 22 March 2023. Biogen's ALS Drug Gets Partial Backing From FDA Panel. Bloomberg News. 25 April 2023 .
  7. Web site: 2023-04-28 . FDA approves drug which helps to slow progression of rare form of MND . 2024-05-16 . www.sheffield.ac.uk . en.
  8. Berdyński M, Miszta P, Safranow K, Andersen PM, Morita M, Filipek S, Żekanowski C, Kuźma-Kozakiewicz M . SOD1 mutations associated with amyotrophic lateral sclerosis analysis of variant severity . Scientific Reports . 12 . 1 . 103 . January 2022 . 34996976 . 8742055 . 10.1038/s41598-021-03891-8 . 2022NatSR..12..103B .
  9. News: Constantino. Annika. 25 April 2023. FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease. CNBC. 25 April 2023. 25 April 2023. https://web.archive.org/web/20230425174051/https://www.cnbc.com/2023/04/25/fda-grants-accelerated-approval-biogen-als-drug.html. live .
  10. News: Constantino. Annika. 22 March 2023. FDA advisors vote against effectiveness of Biogen's ALS drug for rare and aggressive form of the disease. CNBC. 25 April 2023. 10 April 2023. https://web.archive.org/web/20230410174144/https://www.cnbc.com/2023/03/22/fda-advisors-vote-against-effectiveness-of-biogen-als-drug.html. live .
  11. News: Robins. Rebecca. 25 April 2023. F.D.A. Approves Drug for Rare Form of A.L.S.. The New York Times. 25 April 2023. 25 April 2023. https://web.archive.org/web/20230425172634/https://www.nytimes.com/2023/04/25/business/biogen-fda-als-drug.html. live .
  12. New treatment for rare motor neuron disease recommended for approval . European Medicines Agency (EMA) . 23 February 2024 . 24 February 2024.
  13. Web site: Qalsody EPAR . European Medicines Agency (EMA) . 22 February 2024 . 24 February 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  14. Web site: Commission Implementing Decision . 3 June 2024 . 5 June 2024.

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