A second ALS treatment, edaravone, has been newly approved in the United States

Updated May 13, 2022

• In May 2017 the United States Food and Drug Administration approved edaravone (also referred to as Radicava and Radicut) for the treatment of ALS. It’s the second ALS drug to be approved in the US. The first drug, riluzole, was approved more than 20 years ago. Radicava became available in the US in August 2017.
• Radicava is delivered intravenously through infusions over a number of days, with 14 day breaks in between.
• Clinical trial data from the manufacturer, Mitsubishi Tanabe, suggest that Radicava can be effective in improving the condition of people who are early in their ALS progression. A New York Times article describes the drug’s effect as “modest but significant.”
• Radicava may help only a certain segment of people living with ALS, but it is encouraging to see the introduction of a second treatment option in the U.S.
• Now that Radicava has been approved by the FDA, we are hopeful that Mitsubishi Tanabe will take steps for Canadians to be able to access the drug here.
• The hope is that the FDA’s approval of Radicava will help to build further momentum for drug manufacturers to focus on ALS for the development of other therapies, underscoring the need for continued research investment to drive additional scientific discovery.
• On May 12, 2022, FDA announced the approval of an oral formulation of edaravone, also known as oral edaravone, for adults living with ALS. Read more about the decision.
  • In response to CADTH’s call for patient and clinician input on oral edaravone, ALS Canada prepared a patient input submission in April 2022. The ALS clinical community has provided a written submission as well

Edaravone is the generic name for a drug that is marketed as Radicava and sometimes Radicut. Manufactured by the Mitsubishi Tanabe Pharma Corporation, it has been used in Japan since 2001 as a treatment for stroke. Clinical trials testing Radicava as a treatment for ALS have been taking place in Japan since 2006 and in recent years the drug was approved in Japan and South Korea for treating ALS. On May 5, 2017 the U.S. Food and Drug Administration approved Radicava as a treatment for ALS – the first in more than 20 years, and only the second ALS drug to be approved in the US. Radicava became available in the U.S. in August 2017 and the New York Times reports it will cost $145,254 USD per year.

In an earlier clinical trial, Radicava was tested in people in varying stages of ALS progression, and the drug did not show significant slowing of the disease. However, a more recent clinical trial recruited participants who were early in their ALS progression, with milder symptoms and a larger vital capacity (the maximum amount of air a person can expel from their lungs after a maximum inhalation). There were 137 participants in this trial, some of whom received the drug and some of whom received a placebo. According to Mitsubishi Tanabe, after six months the condition of the participants who received the drug declined less than the patients receiving the placebo, as measured using the ALS Functional Rating Scale. This result is described in the New York Times article as “modest but significant.” According to the New York Times, there is no information available as to whether Radicava could help people to live longer with ALS.

Treatment is delivered intravenously every day for 14 straight days, followed by no drug for the next 14 days. This is followed by intravenous treatment over 10 of the next 14 days followed by another 14-day period without the drug. This continues for as long as the treatment is to be administered.

On behalf of ALS societies across the country, ALS Canada is in conversation with Health Canada and with Mitsubishi Tanabe about the status of Radicava and the need for Canadians to have equitable access to this drug through the channels within the healthcare system. Should Radicava be approved by Health Canada, the costs may be different than in the U.S. In the meantime, Canadians interested in learning more about edaravone are advised to connect with their neurologist, who will be able to interpret the clinical information about the drug as it pertains to individual situations.

Radicava may help only a certain segment of people living with ALS, but it is encouraging to see the introduction of a second treatment option in the U.S. As has been seen with cancer and HIV/AIDS, the introduction of new therapies can help to create momentum for drug manufacturers to pursue the development of additional treatment options. We are hopeful the FDA’s approval of Radicava will have a similar impact on the development of ALS therapies – which also underscores the need for continued research investment so the scientific community can continue to pursue discoveries. This in turn will inform the development of additional therapeutic options with even greater potential to make ALS a treatable, not terminal disease.