Amyotrophic Lateral Sclerosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2020

Amyotrophic lateral sclerosis (ALS) is a degenerative motor neuron disorder that leads to progressive muscle wasting and weakness. The typical survival time is three to five years from onset. The FDA’s 2017 approval of Mitsubishi Tanabe’s Radicava resulted in the second approved treatment option for ALS, yet pressing unmet need remains in this arena. The ALS pipeline comprises diverse programs designed to be neuroprotective, to enhance respiratory function, or to restore lost neurons in the spinal cord; however, clinical success in ALS has historically been a formidable challenge. Nonetheless, clinical and 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

• How has Mitsubishi Tanabe’s Radicava been incorporated into the treatment of U.S. ALS patients? What are the key advantages and disadvantages of the product?
• How will the size of the drug-treated ALS population change through 2029?
• What are the key unmet treatment needs in ALS?
• Which emerging therapies have ALS experts most intrigued or optimistic? How would new therapies influence management of ALS patients?
• How are emerging drugs being evaluated, and which are likely to launch by 2029? What commercial impact will they have on the ALS market?

PRODUCT DESCRIPTION

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Scope:

• 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: 6; Phase II/III: 5 Phase II & I/II: 23; coverage of select preclinical and Phase I products.
• Market forecast and alternative market scenarios: Drug-level sales and patient share of ALS therapies in 2029.
• Key companies: Mitsubishi Tanabe Pharma, Biogen, Ionis, Orphazyme, Orion Pharma, AB Science, Neuralstem, BrainStorm Therapeutics.
• Key drugs: Radicava (edaravone), BIIB-067, arimoclomol, levosimendan, masitinib, riluzole, NSI-566, mesenchymal stem cells expanded and induced to secrete neurotrophic factors (MSC-NTF cells).