Tofersen offers potential breakthrough in the fight against amyotrophic lateral sclerosis (ALS)

The Food and Drug Administration (FDA) has given fast-track approval for a new drug, Qalsody, which treats a specific genetic form of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The drug, tofersen, was developed by Biogen Inc. and is designed to help people who have a rare form of the disease, known as SOD1-ALS, which is caused by a mutation in the SOD1 gene. Only 2% of people with ALS have this rare mutation, and the approval of Qalsody is seen as a significant breakthrough in the understanding and treatment of the disease. The drug was approved under the FDA’s accelerated approval pathway, which means that drugs developed for serious conditions that “face an unmet medical need” can be approved relatively quickly.

According to Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University, Qalsody offers hope to those living with SOD1 mutations and has the potential to substantially slow down the genetic form of ALS. In a clinical trial published in The New England Journal of Medicine, Qalsody was found to reduce molecular signs of disease and curb neurodegeneration in the first six months of use.

While Qalsody is only approved for a specific subset of ALS cases, it offers a window into the expanding research and understanding of the disease. Dr. Miller explained that the potential impact of Qalsody for those with SOD1 mutations “is the substantial showing of disease progression and improvements in quality of life.”

Martina H. Wiedau, MD, professor of neurology and the director of the ALS Clinic and Research Center at UCLA Health, said the FDA approval of tofersen “can motivate doctors to conduct genetic testing for SDO1 mutations early and provide this treatment.” Dr. Wiedau, who is unaffiliated with Dr. Miller’s research, pointed out that this study did not select participants who were in early disease stages—some had lived with ALS for a long time. She said “one would assume” that the neurodegenerative process from SOD1-ALS “can be slowed with earlier treatment.”

According to the Centers for Disease Control and Prevention (CDC), there are more than 31,000 people currently living with ALS in the U.S. While Qalsody only addresses a specific population within those totals, Dr. Miller and Dr. Wiedau believe it signals a future where those living with ALS might have more and better options for care. “There is momentum” for the development of new ALS treatments, Dr. Wiedau noted.

Dr. Miller added that Qalsody “can have substantial effects in this genetic subset of ALS” which points to the fact that this disease is a “treatable disorder.” It offers a ray of light to researchers and those living with ALS who don’t have this rare genetic mutation that treatments that will have a “substantial impact on other forms of ALS” are hopefully on the way. The FDA’s approval of Qalsody offers hope to patients with ALS and their families, and researchers continue to work towards finding solutions to this devastating disease.