Abstract
Tofersen (Qalsody™) is an antisense oligonucleotide being developed by Biogen for the treatment of amyotrophic lateral sclerosis (ALS). On 25 April 2023, tofersen was approved in the USA for the treatment of ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This article summarizes the milestones in the development of tofersen leading to this first approval for ALS.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
References
Chen JJ. Overview of current and emerging therapies for amytrophic lateral sclerosis. Am J Manag Care. 2020;26(9 Suppl):S191–7.
Mead RJ, Shan N, Reiser HJ, et al. Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation. Nat Rev Drug Discov. 2023;22(3):185–212.
de Courssou J-BB, Durr A, Adams D, et al. Antisense therapies in neurological diseases. Brain. 2022;145(3):816–31.
Biogen MA Inc. QALSODY (tofersen) injection, for intrathecal use: US prescribing information. 2023. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215887s000lbl.pdf. Accessed 30 May 2023.
Biogen IP. Biogen exercises option with Ionis to develop and commercialize investigational treatment BIIB067 for a subtype of familial amyotrophic lateral sclerosis (ALS) based on positive phase 1 data [media release]. 6 Dec 2018. http://www.biogen.com.
Smith RA, Miller TM, Yamanaka K, et al. Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Investig. 2006;116(8):2290–6.
Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099–110.
Miller TM, Pestronk A, David W, et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013;12(5):435–42.
Miller TM, Cudkowicz ME, Shaw PJ, et al. Safety, PK, PD, and exploratory efficacy in a single and multiple-dose study of a SOD1 antisense oligonucleotide (BIIB067) administered to participants with ALS [abstract no. 6.077 plus poster]. Neurology. 2019;93(5):E533.
Miller T, Cudkowicz M, Shaw PJ, et al. Phase 1–2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2020;383(2):109–19.
Miller TM, Glass JD, Cudkowicz M, et al. Statistical model of the relationship of neurofilament with clinical function in the VALOR phase 3 study of tofersen in adults with SOD1-ALS [abstract no. 32]. Muscle Nerve. 2022;66(S2):S16.
Miller TM, Cudkowicz M, Genge A, et al. Evaluating efficacy and safety of tofersen in adults with SOD1-ALS: results from the phase 3 VALOR trial and open-label extension [abstract no. C05]. In: 33rd International Symposium on ALS/MND. 2022.
Benatar M, Wuu J, Andersen PM, et al. Design of a randomized, placebo-controlled, phase 3 trial of tofersen initiated in clinically presymptomatic SOD1 variant carriers: the ATLAS study. Neurotherapeutics. 2022;19(4):1248–58.
US Food & Drug Administration. FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene [media release]. 25 Apr 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Funding
The preparation of this review was not supported by any external funding.
Authorship and Conflict of interest
During the peer review process, the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Hannah Blair is a salaried employee of Adis International Ltd/Springer Nature and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.
Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability
Not applicable.
Additional information
This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.
Supplementary Information
Below is the link to the electronic supplementary material.
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Blair, H.A. Tofersen: First Approval. Drugs 83, 1039–1043 (2023). https://doi.org/10.1007/s40265-023-01904-6
Published:
Issue Date:
DOI: https://doi.org/10.1007/s40265-023-01904-6