Amyotrophic Lateral Sclerosis | Niche and Rare Pharmacor | G7 | 2015

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease or motor neurone disease (MND), is a degenerative disorder of the nervous system characterized by progressive muscle wasting and weakness. Disease progression is typically rapid—median survival is 3-5 years, and only 20% of patients survive between 5-10 years after symptom onset. At present, the disease-modifying therapy riluzole (Covis Pharmaceuticals’ Rilutek, generics) is the only drug approved specifically for ALS. A majority of ALS patients in both the United States and Europe are treated with riluzole, despite its minimal efficacy (it extends survival by an average of two to three months). ALS experts interviewed acknowledge the limitations of riluzole and emphasize the need for more efficacious disease-modifying therapies for ALS. Thus, there is ample commercial opportunity in the ALS market for therapies that can offer even modest efficacy improvements over riluzole. Experts interviewed also stress the importance of developing ALS biomarkers to advance the development of ALS treatments and expect that targeting multiple pathological mechanisms will be required to achieve a meaningful treatment effect in ALS.

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