Amyotrophic Lateral Sclerosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2024

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 treatments are approved for ALS in the markets under study: Mitsubishi Tanabe Pharma’s Radicava / Radicava ORS (edaravone), riluzole (Advanz Pharma / Sanofi’s Rilutek, other brands, generics), 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 [AMX0035]) from the U.S. market. Thus, substantial clinical and commercial opportunity awaits developers of safe, effective therapies for this disease.

QUESTIONS ANSWERED

• What role do riluzole and Radicava / Radicava ORS play in the treatment of ALS? How is Qalsody being incorporated into medical practice?
• How will the size of the drug-treated ALS population change through 2033?
• What are the key unmet needs in the treatment of ALS?
• Which emerging therapies are ALS experts most intrigued by or optimistic about? How would new therapies influence the management of ALS patients?
• Which therapies for ALS are likely to launch by 2033? What will their commercial impact be?

SCOPE

• Geography: United States and EU5
• Primary research: Six country-specific interviews with thought-leading neurologists; supported by survey data collected for this study
• Epidemiology: Diagnosed prevalent and drug-treated cases of ALS by country, diagnosed prevalence by disease type (familial or sporadic)
• Forecast: Drug-level sales and patient share of key ALS therapies in 2023 and 2033
• Emerging therapies: Coverage of key current and emerging therapies

PRODUCT DESCRIPTION

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

KEY FEATURE

Niche & Rare Disease Landscape & Forecast features a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.