new drug slows the progression of amyotrophic lateral sclerosis. « Medicine in the Library

The FDA has approved Mistake (tofersen) for the treatment of patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce SOD1 protein synthesis. The approval was based on a light reduction of plasma neurofilament (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration.

The green light was granted after observing the results – published in September 2022 on the “New England Journal of Medicine” – obtained at the end of a phase 3 clinical trial of the drug. The study, double-blind and randomized, saw 108 patients enrolled. Tofersen – administered by injection eight injections: three plus five maintenance – proved to be able to reduce the levels of light chain neurofilaments in the plasma of the treated people.

These protein molecules represent a biomarker released into the blood or cerebrospinal fluid in the event of damage to the axons and a degenerative process considered specific to the disease.

Read the full text of the article:
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Timothy M. Miller, M.D., Ph.D., Merit E….. et al.
N Engl J Med 2022; 387:1099-1110 DOI: 10.1056/NEJMoa2204705

(Funded by Biogen; VALOR and OLE ClinicalTrials.gov numbers, NCT02623699 and NCT03070119; EudraCT numbers, 2015-004098-33. opens in new tab and 2016-003225-41.)

FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene – 04/25/2023