Amyotrophic Lateral Sclerosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2023

Amyotrophic lateral sclerosis (ALS) is a progressively debilitating 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, use of limbs, and eventually death. Four treatments are approved for ALS in the markets under study: Mitsubishi Tanabe Pharma’s Radicava (edaravone); riluzole (Advanz Pharma / Sanofi’s Rilutek, other brands, generics); and, most recently, Amylyx Pharmaceuticals’ Relyvrio (sodium phenylbutyrate and taurursodiol [AMX0035]) and Ionis Pharmaceuticals / Biogen’s Qalsody (tofersen). However, pressing unmet need remains. Although the ALS pipeline comprises diverse programs designed to be neuroprotective, enhance respiratory function, or restore lost neurons in the spinal cord, clinical success in ALS has historically been a formidable challenge. Thus, substantial clinical and commercial opportunity awaits developers that can develop a safe, effective therapy for this crippling disease.

QUESTIONS ANSWERED

• What role do riluzole and, in the United States, Radicava play in the treatment of ALS patients? How are Relyvrio and Qalsody being incorporated into medical practice? What are the key advantages and disadvantages of these products?
• How will the size of the drug-treated ALS population change through 2032?
• What are the key unmet needs in the treatment of ALS?
• Which emerging therapies are ALS experts most intrigued or optimistic about? How would new therapies influence the management of ALS patients?
• Which emerging therapies for ALS are likely to launch by 2032? What commercial impact will they have on the 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:

• 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 through 2032
• Emerging therapies: Phase 3/PR: 12; Phase 2: 7; Phase 1: 3

SOLUTION ENHANCEMENT

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.