Turning Setbacks into Strategies: Lessons from RELYVIRO’s journey in ALS
Amyotrophic lateral sclerosis (ALS) is a devastating, rare, aggressive, and complex disease. Affecting approximately 200,000 people globally, it leads to severe disability and, in most cases, death within 2–5 years of diagnosis. In this article and the next we look at the recent developments for new ALS treatments. To begin with we focus on what many had hoped would be a breakthrough: the story of RELYVIRO (AMX0035). Its development was marked by successes and setbacks, and ultimately resulted in failure with the product being withdrawn from the market by its manufacturer Amylyx earlier this year. Our purpose here is to learn from this in order to inform future drug development in rare diseases. In the second article to follow, we will look at the late-stage ALS pipeline and see how sponsors are responding to these challenges and the promise they hold for improving the lives of ALS patients. ALS: a devastating disease ALS is a progressive neurodegenerative disease that affects mot...
