Biogen

The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen’s tofersen, now named Qalsody, for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The decision, which marks the first conditional approval for ALS in the country, comes about eight…

The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Biogen for approval of the company’s experimental therapy tofersen to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The FDA has granted the application accelerated review and a decision…

Early use of Biogen’s tofersen significantly slows disability progression, as well as a decline in lung function, muscle strength, and quality of life in amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, compared with a six-month delay in starting treatment. These benefits were accompanied by pronounced and sustained…

Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…

Tofersen, an experimental treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, failed to significantly slow the rate of disease progression relative to a placebo in a Phase 3 trial. These top-line findings mean that the VALOR study (NCT02623699), which is testing tofersen in…

The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…

People with very rapidly progressing familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene may gain early access to Biogen’s experimental therapy tofersen by mid-July, the company announced. This marks the first part of Biogen’s planned access program for tofersen, and will begin after patients initially assigned…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. A new clinical trial will seek to determine the optimal timing to begin treatment with the investigational medication tofersen in people…

Note: This story was updated on April 16, 2021, to note that Scribe’s founding year was 2018, not 2017. Scribe Therapeutics has raised $100 million in funding to develop CRISPR-based gene editing tools and advance its pipeline of therapies for neurodegenerative diseases, such as…