Tofersen

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…

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…

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 European Medicines Agency (EMA) has agreed to review an application seeking the approval of tofersen in treating amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, the therapy’s developer, Biogen, reported. “Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need…

Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…

The U.S. Food and Drug Administration (FDA) is extending by three months its review of tofersen, Biogen’s investigational treatment for forms of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. Earlier this summer, the FDA granted the application priority review, with a decision expected no…

Early treatment with Biogen’s experimental therapy tofersen slows disease progression in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations, compared with patients who started treatment after a six-month delay, according to findings from a Phase 3 trial and its open-label extension study. These benefits…

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…

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…