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.

Questions Answered in This Report:

  • Amyotrophic Lateral Sclerosis (ALS) is a rare disease affecting only a small percentage of the population. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) diagnosed prevalent ALS population? What percentage of the ALS population has a familial form of the disease? How will the size of the ALS population change over the 2014-2024 forecast period?

  • Although ALS is one of the few neurological disorders for which a disease-modifying therapy is available, a high level of unmet need for new ALS treatments remains. For example, improved disease-modifying therapies are sorely needed, as are therapies that improve function, and, ultimately, therapies that provide a cure. What are interviewed experts’ attitudes towards each of these unmet needs? Are there any treatments in clinical development that are likely to address these needs?

  • The ALS therapeutic pipeline is robust and diverse, featuring different potential therapeutic approaches to treating the disease. Which emerging therapies do experts view as most promising? What are the key primary and secondary clinical end points on which new therapies are evaluated? Which emerging therapies are likely to launch by 2024? How would therapies expected to launch be incorporated into the ALS treatment algorithm, and in what percentage of patients would such therapies be used?


Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with ALS neurologists.

Epidemiology: Diagnosed prevalence, diagnosed prevalence by disease type (familial or sporadic ALS), diagnosed prevalence by comorbid frontotemporal dementia.

Emerging therapies: Phase III: 3; Phase II: 18; Phase I/II: 2; coverage of select preclinical and Phase I products.

Author(s): Matthew R. Mackenzie, M.A.
Prachi Vora, M.P.H.

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