Amyotrophic Lateral Sclerosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2017

Amyotrophic lateral sclerosis (ALS) is a degenerative disorder of motor neurons that leads to progressive muscle wasting and weakness, with a typical survival time of three to five years from onset. Riluzole (Concordia Health/Sanofi’s Rilutek, generics) is the only approved disease-modifying therapy (DMT) for ALS, although interviewed experts consider the drug’s efficacy to be modest. Experts emphasize the unmet need for additional, more effective ALS DMTs, as well as new symptomatic drugs, and biomarkers to aid in diagnosis, prognosis, and pharmacodynamics. The ALS clinical pipeline is robust and diverse, comprising late-stage programs aimed at enhancing muscle function, immune modulation, and neuroprotection, as well as mid– or early-stage gene therapy or gene silencing approaches, stem-cell therapies, and immune-targeting agents—and experts contend that regimens combining multiple mechanisms of action could hold significant clinical promise in the management of ALS. Thus, considerable commercial opportunity exists for developers that can overcome the scientific and clinical hurdles blocking the path to regulatory approval for the treatment of this devastating disease.

Questions Answered:

  • Amyotrophic Lateral Sclerosis (ALS) is a rare disease affecting only a small percentage of the population. How large is diagnosed prevalent ALS population in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom)? What percentage of the ALS population has a familial versus sporadic form of the disease? What percentage is afflicted with comorbid frontotemporal dementia? How will the size of the ALS population change over the 2016-2026 forecast period? What percentage of diagnosed ALS patients receives drug treatment?
  • With only a single drug approved to prolong survival and maintain function, a high level of unmet need for new ALS treatments exists. What are the key drug targets emerging from basic and clinical research in ALS? What are the most pressing gaps in treatment identified by interviewed ALS experts? How well will emerging therapies in the ALS pipeline address key unmet treatment needs?
  • The ALS pipeline comprises therapies with diverse mechanisms of action and technologies. How are emerging therapies evaluated in clinical trials, and which programs do interviewed experts consider the most promising? Which emerging therapies are likely to launch by 2026, and what clinical and commercial impact will they have?


  • Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.
  • Primary research: Six 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/PR: 3; Phase II: 8; Phase I/II: 2; coverage of select preclinical and Phase I products.
  • Market forecast and alternative market scenarios: Drug-level sales and patient share of ALS therapies in 2026.
  • Key companies: Cytokinetics, Mitsubishi Tanabe Pharma, ​​AB Science, Neuralstem, BrainStorm Therapeutics, Neuraltus Pharmaceuticals, Biogen, Ionis Pharmaceuticals.
  • Key drugs: Tirasemtiv, edaravone, masitinib, riluzole, NSI-566, NurOwn, NP-001.
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