Tofersen

Tofersen is indicated to treat people with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).

Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen.{{cite news|last=Liu|first=Angus|date=1 May 2019|title=Biogen's antisense ALS drug shows promise in early clinical trial|url=https://www.fiercebiotech.com/research/biogen-s-antisense-als-drug-shows-promise-early-clinical-study|work=FierceBiotech|access-date=25 April 2023|archive-date=2 February 2023|archive-url=https://web.archive.org/web/20230202115409/https://www.fiercebiotech.com/research/biogen-s-antisense-als-drug-shows-promise-early-clinical-study|url-status=live }}{{cite news |last=Langreth|first=Robert|date=22 March 2023|title=Biogen's ALS Drug Gets Partial Backing From FDA Panel|url=https://www.bloomberg.com/news/articles/2023-03-22/biogen-s-als-drug-tofersen-gets-partial-backing-from-fda-panel|work=Bloomberg News|access-date=25 April 2023 }}

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.{{Cite web |date=28 April 2023 |title=FDA approves drug which helps to slow progression of rare form of MND |url=https://www.sheffield.ac.uk/news/fda-approves-drug-which-helps-slow-progression-rare-form-mnd |access-date=16 May 2024 |website=www.sheffield.ac.uk |language=en}}

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

Only around 1-2% of ALS cases diagnosed in the United States each year carry the specific SOD1 mutation targeted by the drug.{{cite journal | vauthors = Berdyński M, Miszta P, Safranow K, Andersen PM, Morita M, Filipek S, Żekanowski C, Kuźma-Kozakiewicz M | title = SOD1 mutations associated with amyotrophic lateral sclerosis analysis of variant severity | journal = Scientific Reports | volume = 12 | issue = 1 | pages = 103 | date = January 2022 | pmid = 34996976 | pmc = 8742055 | doi = 10.1038/s41598-021-03891-8 | bibcode = 2022NatSR..12..103B }} 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.{{cite news|last=Constantino|first=Annika|date=25 April 2023|title=FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease|url=https://www.cnbc.com/2023/04/25/fda-grants-accelerated-approval-biogen-als-drug.html|work=CNBC|access-date=25 April 2023|archive-date=25 April 2023|archive-url=https://web.archive.org/web/20230425174051/https://www.cnbc.com/2023/04/25/fda-grants-accelerated-approval-biogen-als-drug.html|url-status=live }}{{cite news|last=Constantino|first=Annika|date=22 March 2023|title=FDA advisors vote against effectiveness of Biogen's ALS drug for rare and aggressive form of the disease|url=https://www.cnbc.com/2023/03/22/fda-advisors-vote-against-effectiveness-of-biogen-als-drug.html|work=CNBC|access-date=25 April 2023|archive-date=10 April 2023|archive-url=https://web.archive.org/web/20230410174144/https://www.cnbc.com/2023/03/22/fda-advisors-vote-against-effectiveness-of-biogen-als-drug.html|url-status=live }}{{cite news|last=Robins|first=Rebecca|date=25 April 2023|title=F.D.A. Approves Drug for Rare Form of A.L.S.|url=https://www.nytimes.com/2023/04/25/business/biogen-fda-als-drug.html|work=The New York Times|access-date=25 April 2023|archive-date=25 April 2023|archive-url=https://web.archive.org/web/20230425172634/https://www.nytimes.com/2023/04/25/business/biogen-fda-als-drug.html|url-status=live }}

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.{{cite press release | title=New treatment for rare motor neuron disease recommended for approval | website=European Medicines Agency (EMA) | date=23 February 2024 | url=https://www.ema.europa.eu/en/news/new-treatment-rare-motor-neurone-disease-recommended-approval | access-date=24 February 2024}} The applicant for this medicinal product is Biogen Netherlands B.V.{{cite web | title=Qalsody EPAR | website=European Medicines Agency (EMA) | date=22 February 2024 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/qalsody | access-date=24 February 2024}} Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Tofersen was approved for medical use in the European Union in May 2024.{{cite web | title=Qalsody PI | website=Union Register of medicinal products | date=3 June 2024 | url=https://ec.europa.eu/health/documents/community-register/html/h1783.htm | access-date=7 September 2024}}

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Category:Amyotrophic lateral sclerosis

Category:Therapeutic gene modulation

Category:Orphan drugs