MAY 11, 2017 6:20 AM PDT

First ALS Drug Approved in Over 2 Decades


For the first time in over 20 years, the US Food and Drug Administration approved a new drug for the treatment of a disease commonly known as Lou Gehrig’s disease.

Known formally as amyotrophic lateral sclerosis (ALS), Lou Gehrig's disease is a fatal condition caused by the gradual breakdown and death of motor neurons in the spinal cord. All muscles under voluntary control are affected, causing muscle weakness and severe impairments in daily functions. There is no known cure and most ALS patients die from respiratory failure within 3 to 5 years from the onset of symptoms.

The new drug is known as Radicava or edaravone, and it was developed by Japanese Mitsubishi Tanabe Pharma. Trials for the drug conducted in Japan showed the drug slowed the progression of the disease. The 2015 phase 3 data from a six-month placebo-controlled study found the drug contributed to a 33 percent reduction in physical decline in ALS patients. So while the drug can’t reverse the trajectory of ALS, it could give patients just a little more time and freedom.

Interestingly, the FDA’s approval of the drug was based solely on data collected from the Japanese trial. These results were so compelling that the FDA worked with the company to file for US approval.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

Radicava is administered intravenously, with each infusion estimated to cost $1,086, and a year’s treatment costing $145,524 without assistance.

Prior to radicava, the 12-15,000 ALS patients had access to only one other approved ALS drug, riluzole by Sanofi. Riluzole was approved for ALS in 1995. Similar to radicava, the drug slows the progression of the disease, delaying the onset of ventilator-dependence and tracheotomy in some patients. The drug is estimated to prolong survival by two to three months.

For ALS patients and advocates, the approval of radicava marks a significant milestone. "We thank the FDA and MT Pharma for working together to expedite the approval of the first new ALS-specific treatment in decades," said ALS Association President Barbara Newhouse. "We hope (the) announcement signals the beginning of a new chapter in the fight against this terrible disease." The new drug is expected to come on the US market in August 2017.

Additional source: CNN

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About the Author
  • I am a human geneticist, passionate about telling stories to make science more engaging and approachable. Find more of my writing at the Hopkins BioMedical Odyssey blog and at
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