Changing America

FDA panel rejects ALS drug partly funded by Ice Bucket Challenge

Advocates were hopeful the drug could help thousands of people with the fatal disease. 
FDA headquarters
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Story at a glance

  • A Food and Drug Administration panel voted to reject a new drug meant to treat ALS, to the disappointment of patients and advocacy groups backing the drug.

  • ALS, or Amyotrophic Lateral Sclerosis, is a progressive neurodegenerative disease that impacts a person’s ability to move their muscles eventually resulting in death.

  • There is no cure for ALS, and some patients and advocates are eagerly waiting for the drug’s approval despite there being limited research showing it works.

A Food and Drug Administration (FDA) panel narrowly voted against recommending a new drug meant to treat the neurodegenerative disease ALS, dealing a harsh blow for some patients and advocate groups.  

The Peripheral and Central Nervous System Drugs Advisory Committee voted 4-6 after meeting all day on Wednesday to discuss the new drug and whether there was sufficient evidence to prove it was effective.  

The committee stated the company’s clinical study data did not establish that it was effective against the disease.  


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ALS, which stands for Amyotrophic Lateral Sclerosis and is also known as Lou Gehrig’s Disease, is a fatal neurodegenerative disease with no known cure. The illness causes a person to progressively lose control over their muscles and eventually lose the ability to walk, talk and breathe.  

Between 12,000 and 15,000 people in the United States have the rare disease, according to the Centers for Disease Control and Prevention, with most passing away from the condition between two to five years after diagnosis.  

The drug was created by the Massachusetts-based company Amylyx Pharmaceuticals and is meant to slow the progression of the disease by blocking motor neurons, or the cells that send electrical signals from the brain to muscles, from dying early. 

In response to the FDA’s decision, Amylyx said it is confident in the trial data and the benefits of the drug as a treatment option for ALS, according to Reuters.  

Advocacy groups I AM ALS and the ALS Association funded a portion of Amylyx’s study partly with funds from the 2014 Ice Bucket Challenge, according to NBC. 

Typically, the FDA requires at least two large, late-stage studies to approve a drug, according to ABC. But for life-threatening diseases like cancer, a single study with early and promising results have been accepted, the outlet added.  


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