Tofersen

Excerpt

Tofersen, a novel antisense oligonucleotide (ASO) drug, received accelerated approval from the U.S. Food and Drug Administration (FDA) in April 2023 for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a progressive, fatal neurodegenerative disorder affecting motor neurons in the brain and spinal cord, leading to muscle weakness, paralysis, and eventual death. Tofersen is specifically indicated for ALS patients with a superoxide dismutase 1 (SOD1) gene mutation. The mutant SOD1 protein resulting from this gene mutation gains a toxic function and significantly increases SOD1 messenger RNA (mRNA) levels and protein in the cerebrospinal fluid (CSF). This activity provides comprehensive information about Tofersen's indications, contraindications, mechanism of action, adverse event profile, monitoring, and relevant interactions relevant to patients and healthcare team members involved in treating patients with ALS and related conditions.