Despite the substantial unmet need for ALS treatment, an incomplete understanding of disease etiology has contributed to the high rate of clinical trial failure. 

Only three drugs are approved for ALS, but none of them offer substantial benefit in slowing of functional decline or survival time. The usage of biomarkers such as neurofilament light chain levels as a surrogate endpoint is becoming more common, sometimes as a surrogate endpoint for the FDA accelerated approvals pathway, though many drugs are unable to demonstrate statistically significant improvements on this endpoint either.

Given the high unmet need for effective treatment, patient advocacy groups are heavily involved in both the drug development and regulatory processes. This lack of treatment has also led to a high degree of regulatory flexibility, and many companies choose to leverage the accelerated approvals pathway so patients may access the drug while confirmatory trials are ongoing.

This Datamonitor Healthcare report contains a Disease Analysis module.