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.
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.