Amyotrophic Lateral Sclerosis – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

Amyotrophic lateral sclerosis (ALS) is a disorder of the central nervous system characterized by motor neuron degeneration. ALS causes progressive weakness and atrophy of the muscles and loss of voluntary functions, including breathing, swallowing, and use of limbs, and eventually death. Three disease-modifying therapies are approved for ALS in the markets under study: riluzole (Sanofi’s Rilutek, other brands, generics), Mitsubishi Tanabe Pharma’s Radicava / Radicava ORS (edaravone), and Ionis Pharmaceuticals / Biogen’s Qalsody (tofersen). Although the ALS pipeline comprises diverse drugs designed to enhance respiratory function, be neuroprotective, or restore lost neurons in the spinal cord, clinical success has been a formidable challenge, as evidenced by the late 2023 withdrawal of the BLA for BrainStorm Cell Therapeutics’ NurOwn and the 2024 withdrawal of Amylyx Pharmaceuticals’ Relyvrio (sodium phenylbutyrate and taurursodiol FDC) from the U.S. market. Thus, substantial clinical and commercial opportunity awaits developers of safe, effective therapies for this disease.

Questions answered

• How will the size of the drug-treated ALS population change through 2034?
• What role do riluzole and Radicava / Radicava ORS play in the treatment of ALS? How is Qalsody being incorporated into medical practice?
• Which therapies for ALS are likely to launch by 2034? What will their commercial impact be?
• Which emerging therapies are ALS experts most intrigued by or optimistic about? How would new therapies influence the management of ALS patients?
• What are the key unmet needs in the treatment of ALS?

Content highlights

• Publication date: November 2025
• Geography: United States and EU5
• Primary research: Country-specific interviews with thought-leading neurologists; survey data collected for this and other Clarivate research
• Epidemiology: Diagnosed prevalent and drug-treated cases of ALS by country and subtype (i.e., familial and sporadic)
• Forecast: Drug-level sales and patient share of key ALS therapies in 2024 and 2034
• Drug treatments: Coverage of key current and late-phase emerging therapies

Product description

Niche & Rare Disease Landscape & Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.