ALS Therapeutics Market to Near $2B by 2035
The global market for amyotrophic lateral sclerosis (ALS) therapeutics is projected to grow from $1.05 billion in 2025 to $1.96 billion by 2034. The market is expected to expand at a compound annual growth rate of 11.5%, driven by the development of new therapies and an increasing prevalence of the disease worldwide.
- The first drug for ALS, Riluzole, was approved in 1995 and is thought to work by reducing levels of the neurotransmitter glutamate. It was the only approved treatment for 22 years until the approval of Edaravone (Radicava) in 2017, a free-radical scavenger that aims to prevent oxidative stress damage to motor neurons. - An estimated 5-10% of ALS cases are "familial," meaning they are inherited, while the rest are considered "sporadic" with no clear cause. More than 40 genes have been linked to the disease; mutations in four genes—*C9orf72*, *SOD1*, *TARDBP*, and *FUS*—account for the majority of familial cases. - The approval of Tofersen (Qalsody) in 2023 marked a significant step towards precision medicine for ALS. It is an antisense oligonucleotide (ASO) therapy that specifically targets the RNA produced from a mutated *SOD1* gene, preventing the creation of toxic proteins. This approach is only effective for the 2% of ALS patients with this specific mutation. - The path to new treatments is challenging; in April 2024, the drug Relyvrio was voluntarily removed from the market after its Phase 3 clinical trial failed to meet its goals, despite showing promise in an earlier Phase 2 study. - Globally, the incidence of ALS (new cases) ranges from 0.26 per 100,000 people in Ecuador to 23.46 per 100,000 in a region of Japan, while the prevalence (total cases) can be as high as 11.8 per 100,000 in the United States. The total number of worldwide cases is projected to increase from around 223,000 in 2015 to over 376,000 by 2040. - The clinical pipeline includes a variety of next-generation approaches. These include gene therapies using viral vectors to deliver therapeutic antibodies directly to the central nervous system, stem cell therapies aimed at supporting damaged neurons, and molecules designed to correct errors in RNA processing.
