FDA Accepts Pharma’s New Drug Application for ALS Therapy Edaravone

FDA Accepts Pharma’s New Drug Application for ALS Therapy Edaravone

The U.S. U.S. Food and Drug Administration (FDA) has accepted Mitsubishi Tanabe Pharma’s new drug application (NDA) for edaravone as a treatment for amyotrophic lateral sclerosis (ALS).

A decision regarding the intravenous treatment is expected by June 16, 2017, based on the U.S. Prescription Drug User Fee Act. If it is approved, the new medicine will be marketed under the brand name Radicava through the newly formed company MT Pharma America.

“This is an important milestone for Mitsubishi Tanabe Pharma and for the U.S. ALS community,” Dr. Joseph M. Palumbo, MD, vice president and head of clinical research at Mitsubishi Tanabe Pharma Development America, said in a press release.

“There is an urgent need for new treatment options in ALS and we are now an important step closer to potentially making that a reality. We look forward to working with the FDA as part of the review process,” Palumbo said.

The edaravone NDA is supported by a clinical research program in Japanese ALS patients. In 2015, edaravone was approved for use as an ALS treatment in Japan and South Korea. It was also granted orphan drug designation by the FDA and the European Commission. The FDA has not yet approved edaravone for any indication.

Edaravone is a potent antioxidant free radical scavenger that is believed to relieve the effects of oxidative stress.

Oxidative stress is a probable factor for the onset and progression of ALS, and it is common to find consistent increases in oxidative stress biomarkers in patients. It results from an imbalance between the production of free radicals and the body’s ability to counteract or detoxify their harmful effects.

ALS, also called Lou Gehrig’s disease, attacks nerve cells in the brain and the spinal cord responsible for controlling voluntary muscles, affecting a patients ability to move, speak, eat, or breathe. It is one of the most well-known neuromuscular diseases in the world, affecting nearly 2 in every 100,000 people. It is estimated that 5 to 10 percent of all known ALS cases are inherited, but the cause of most ALS cases is not well understood.

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18 comments

  1. Linda gibson says:

    What will this drug do /how will it help those people with ALS already on vents and unable to move -yet that are still very mentally aware of everything and very healthy?

  2. Stephen Fazio says:

    I’m undergoing diagnosis but Ithe is progressing to the point it can’t be ignored. How can this medication help me?

    • Tim Bossie says:

      As the article says, “Edaravone is a potent antioxidant free radical scavenger that is believed to relieve the effects of oxidative stress.” This means the this treatment can help to ability to counteract or detoxify the effects of this stress.

  3. Dorothy Ziminski says:

    If someone is willing and able to travel to Japan is there any groups/hospitals/organizations we can contact us to help with setting up treatment when we get there?

    • Tim Bossie says:

      Hi Dorothy! We do not know the answer to that, but maybe it would be worth contacting the ALS Association? They might be able to help with that.

    • JD Morrissey says:

      I just got back from Japan with my girlfriend who speaks japanese and we were able to get the medication for her uncle and family friend. The medication there was only 2000yen a vile which comes to less that 20usd. We have a way to do it if you’re still looking for help.

      • Chris clark says:

        My brother has ALS and I would like to get him some radicut in Japan asap. Could you contact me or send me detatils of what I need to do to accomplish the trip. Thanks

        • erica says:

          Hi, My girlfriend is back stateside and she can help you better its easier for her to give you the load down over the phone if you want to give her a call her number is 206-484-9877 or email her at eri.irby@gmail.com and she’s happy to help anyone with ALS!

  4. Michelle Geoffrion-Vinci says:

    Hi Tim – My husband was diagnosed last fall. How do we determine the presence and increase of oxidative stress biomarkers in his system? Thanks!

    • Tim Bossie says:

      Hi Michelle, I am sorry to hear that your husband has been given a diagnosis of ALS. If you are concerned about increases of oxidative stress you will have to talk with your doctor about this as it is something that happens at the cellular level.

  5. DR. NEHAL says:

    MY sister was diagnosed as ALS 2 years ago . she is 36 years old. is Edaravon efficient?? i read that it prevent progression of the disease by 4 % is it true?

    • Tim Bossie says:

      Hello Dr Nehal. We are sorry to hear about your sister. ALS is such a devastating disease. Sadly, there isn’t a lot of treatment available for it right now. We are not sure about any percentages involved with edaravon and its ability to prevent progression.

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