USA: FDA Confirms the Guidelines in the Development of ALS Drugs

The U.S. Food and Drug Administration (FDA) has issued its final guidance to support sponsors developing drugs and biological products intended for the treatment of amyotrophic lateral sclerosis (ALS). This document is designed to address the unique considerations involved in ALS clinical development programs.

Originally released as a draft in February 2018, the guidance has now been refined and expanded, incorporating updates and clarifications to improve usability and flexibility during drug development. Its primary focus is to highlight clinical trial design elements and development issues that are specific to ALS.

While the guidance outlines major recommendations, it does not establish legally binding requirements. Instead, it offers a framework intended to help sponsors design scientifically sound and efficient ALS clinical trials that adequately evaluate safety and effectiveness.