The U.S. FDA approved RADICAVA IV formulation on May 5, 2017 as a treatment for ALS. In May 2022 the FDA approved an oral formulation of edaravone for the treatment of ALS. Find answers to frequently asked questions below.
- What does edaravone do?
- How does edaravone work?
- What have researchers learned about edaravone so far?
- How should I decide whether edaravone is right for me?
- How is RADICAVA IV administered?
- How can people with ALS Access Radicava IV?
- What is oral edaravone?
- I am currently taking edaravone intravenously. Should I start taking the oral version?
- I have a feeding tube. Can I use oral edarvone?
- Will IV edaravone continue to be available?
- Will oral edaravone be covered by my insurance?
- If I am considering edaravone, where can I get more information?
What does edaravone do?
Edaravone is intended to slow the progression of ALS by preventing nerve damage.
How does edaravone work?
Scientists believe edaravone protects neurons by capturing unstable molecules that damage cells. These unstable molecules, called reactive oxygen species, form when cells consume energy, and scientists think abnormal buildup of these molecules contributes to the neurodegeneration seen in ALS.
What have researchers learned about edaravone so far?
Edaravone IV (RADICAVA®)
The U.S. FDA approved RADICAVA IV formulation on May 5, 2017, as a treatment for ALS.
In 2015, edaravone IV was approved for the treatment of ALS in Japan in South Korea. As of today, edaravone IV is approved in Canada (October 2018), Switzerland (January 2019), China (July 2019), Indonesia (July 2020), and Thailand (April 2021).
Here is a brief overview of the clinical trial data gathered around RADICAVA IV formulation. The evidence for efficacy is mixed and it may be that RADICAVA IV is only efficacious in certain patients, especially given the wide heterogeneity of ALS.
Originally, edaravone was approved in Japan to treat stroke. A phase III double-blind placebo-controlled study (MCI186-J16) was conducted in people living with ALS to confirm safety and efficacy of edaravone. This study showed that edaravone did not significantly differ from placebo using the ALS Functional Rating Scale Revised (ALSFRS-R). However, post-hoc analysis of that study suggested that people living with ALS that had greater baseline functionality might have benefited, so investigators conducted another double-blind placebo-controlled phase III trial (MCI186-19) with that sub-group of people living with ALS. Results demonstrated that people living with ALS that had a forced vital capacity of >80% and had disease less than 2 years showed the greatest benefit with a statistically significant improvement in the ALSFRS-R.
Further analysis of the MCI186-19 study published in Muscle Nerve journal in 2020 explored the long-term efficacy of edaravone (IV administered) during the open-label extension period of that study. This post-hoc study showed that the change in ALSFRS-R score remained significantly lower for the edaravone treatment group than for the placebo group for up to 48 weeks. In contrast, a recent cohort study (a different type of study than the double-blind placebo-controlled phase III studies) was conducted in ALS referral centers associated with the German Motor Neuron Disease Network. This study used a technique called propensity score matching to compare patients receiving edaravone to similar patients who were not receiving edaravone and found that edaravone treatment was not associated with any disease modifying benefit.
Oral edaravone (MT-1186)
Results of the phase 3 clinical trial evaluating the safety and tolerability of the oral suspension over 24-weeks in people with ALS was announced by MPTA at the 32rd International Symposium of ALS/MND on December 9, 2021.
The phase 3 study was a global, multi-center, open-label study that enrolled 185 people with ALS across 50 sites in the U.S., Canada, Europe, and Japan. The study evaluated safety and tolerability at the 24- and 48-week timepoints. At the conference, the 24-week data was presented. No serious adverse events considered to be treatment-related by investigators were reported.
There is also an ongoing long-term safety extension study of oral edaravone for up to 96 weeks, which will report on the 48-week and 96-week timepoints.
Learn more about the phase 3 oral edaravone development program is available at ClinicalTrials.gov (NCT04165824 and NCT04577404).
How should I decide whether edaravone is right for me?
Talk to your doctor or multidisciplinary care team about your treatment options.
How is RADICAVA IV administered?
People with ALS take RADICAVA IV through an IV – the drug gets injected into their vein over the course of 1 hour. Your healthcare provider will tell you how often you will receive RADICAVA and for how long you will stay on RADICAVA.
Learn more here:
The version under FDA review today is an oral suspension. It is a powder that mixes into drink that could be administered by mouth or via feeding tube.
How can people with ALS Access Radicava IV?
We encourage you to speak to your physician about whether or not RADICAVA IV is right for you. More information about Radicava IV here.
What is oral edaravone?
Radicava ORS is an oral formulation of edaravone – a therapy first approved for intravenous use to treat ALS in 2017.
I am currently taking edaravone intravenously. Should I start taking the oral version?
The decision whether to take edaravone and which formulation is right for you should be made in consultation with your doctor or multidisciplinary care team. As you and your doctors evaluate whether oral Radicava is appropriate, you may consider the impact of possibly eliminating infusion (and the associated potential health impacts, travel and costs).
I have a feeding tube. Can I use oral edarvone?
Radivaca ORS has been approved for use with a feeding tube.
Will IV edaravone continue to be available?
Yes, both forms of edaravone will continue to be an option for people ling with ALS.
Will oral edaravone be covered by my insurance?
Drug coverage decisions will be made by Medicare, the Veterans Administration and commercial private insurers. We are urging all insurers, including Medicare, the VA and commercial health insurance companies to cover oral edaravone.
If I am considering edaravone, where can I get more information?
MTPA provides the JourneyMate Support Program™ which offers educational support and resources for patients who are considering or have already been prescribed an MTPA product. Experienced program team members are trained to address patient and caregiver educational needs and provide them with personalized answers and resources for living with ALS. This program is here to supplement the resources that a doctor provides. For more information, call 1-866-684-7737 or visit radicava.com/journeymate.
Learn more about RADICAVA IV and oral edaravone:
MTPA’s website here.
Recent MTPA press releases:
- Mitsubishi Tanabe Pharma America Announces Initiation of Open-Label Extension Study of Oral Edaravone in ALS
- Mitsubishi Tanabe Pharma America Announces Results from the Global Phase 3 Safety Study of Investigational Oral Edaravone in the Treatment of ALS
- Mitsubishi Tanabe Pharma America Announces FDA Acceptance of New Drug Application (NDA) for Oral Edaravone Formulation for the Treatment of ALS
NEALS database for information on oral edaravone clinical trials:
Read past ALS Association blogs featuring RADICAVA IV or oral edaravone here:
