Melanie Lendnal, Senior Vice President of Policy & Advocacy at The ALS Association, discusses the Accelerated Approval of Qalsody (tofersen) for patients with amyotrophic lateral sclerosis (ALS).
ALS is a rare motor neuron disease leading to problems with muscle control and movement. There are various types of ALS that are distinguished by symptoms and, in some cases, genetic cause. Early symptoms may include muscle twitching, cramping, stiffness, or weakness, slurred speech, and/or difficulty chewing or swallowing. As the disease progresses, people may become weaker and are eventually wheelchair-dependent. Most people with ALS have a sporadic form of ALS. It is believed that these cases are caused by an interaction between genetic and environmental factors. About 10% of the people with ALS have at least one relative with the disease and are said to have a familial form of the disease. Familial ALS may be caused by genetic changes in any one of several genes and the pattern of inheritance varies depending on the gene involved.
The ALS Association’s mission is to make ALS liveable and cure it. The organization has three areas of focus: care services, research, and advocacy. Care services work to provide a support person for patients seeking care throughout their entire journey. The organization’s advocacy work is rooted in ensuring the best and most efficient policies are being passed at all levels of government.
Accelerated Approval Treatment for ALS
Ms. Lendnal explains how much progress has been made in the ALS community, with many treatments approved by the FDA that slow the progression of the disease. Her talk at the 2025 World Orphan Drug Congress focused on a treatment that has been granted Accelerated Approval of tofersen that targets the SOD1 form of genetic ALS. Tolfersen is an antisense oligonucleotide that binds directly to specific mRNA and reduces the amount of neurofilament light chain in the blood. In clinical trials the treatment has shown to not just slow disease progression, but allow for the regaining of function in patients with ALS.
For more information on ALS and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/