Tofersen is an investigational antisense medicine that is designed to inhibit the production of superoxide dismutase 1 (SOD1) and is currently being studied in presymptomatic adult carriers with a SOD1 mutation, a well understood genetic cause of amyotrophic lateral sclerosis (ALS).
Tofersen is also approved in the U.S. for the treatment of patients with SOD1 ALS under the brand name QALSODY® (tofersen). Please see the full Prescribing Information.
About Superoxide Dismutase 1 (SOD1) Amyotrophic Lateral Sclerosis (ALS)
SOD1-ALS is a rare, fatal, neurodegenerative disorder caused by a mutation in the SOD1 gene leading to a progressive loss of motor neurons. As a result, people with SOD1-ALS experience increasing muscle weakness, loss of movement, difficulty breathing and swallowing and eventually succumb to the disease. SOD1-ALS is diagnosed in approximately 2% of all ALS cases, impacting about 330 people in the United States. Approximately 5-10% of people with ALS have a genetic form of the disease of whom about 20% will have a mutation of the SOD1 gene; however, they may not have a known family history of the disease.
Safety and efficacy have not been evaluated by any regulatory authorities for the SOD1-ALS indication described.