What does the drug do?
Edaravone was originally developed to treat acute ischemic stroke, however it was then tested in people with MND. One theory about why motor neurones die in MND is that they are affected by oxidative stress. Cell’s normal metabolic processes can cause oxidative stress. This can result in cellular damage resulting in excessive accumulation of free radiations. Edaravone acts as a ‘free radical scavenger’ helping to reduce the effects of oxidative stress and hopefully slow the progression of MND.
Phase 3 (intravenous formulation)
The first Phase 3 clinical trial was for an intravenous formulation (straight into the bloodstream) of edaravone (Radicava). It was found to provide no overall benefit in people with MND, but showed an improvement in breathing function in a subgroup of people with an early form of the disease. Two studies have been conducted in this subgroup of patients. Results of the second study, announced at the 27th International Symposium on ALS/MND in 2016, revealed that after 24-week intervention patients showed some signs of beneficial effect. It has been estimated that approximately 7% of people with MND were eligible to take part in the clinical trials, showing improvement in only a small amount of people.
Edaravone (Radicava) is currently available in Japan, South Korea, the U.S, Canada, Switzerland, and China.
The Phase 3 clinical trial testing an oral suspension formulation of edaravone (MT-1186) was launched in November 2019, but was paused in March 2020 due to the COVID-19 pandemic. The trial recruited 185 participants between 18 to 75 years. Interim data presented at the 32nd International Symposium on ALS/MND showed that it was safe and well tolerated. Furthermore, it was found that the decline in ALS Functional Rating Scale-Revised (ALSFRS-R) was comparable to the non-oral form of edaravone (Radicava). You can find out more about this trial here.
A Phase 3b trial is aiming to recruit 380 participants to evaluate and compare the efficacy of two dosing regimens of oral edaravone in people with ALS based on the change in ALS Functional Rating Scale-Revised (ALSFRS-R) score from baseline up to Week 48. You can read more about the trial here.
This trial is recruiting in the US and Japan only.
May 2022 - The FDA announced that oral edaravone (Radicava-ORS) has been approved for use in the US. You can read more here.
Jan 2022 - Mitsubishi Tanabe Pharma America announced FDA acceptance of a New Drug Application. You can read more about it here.
Dec 2021 - Mitsubishi Tanabe Pharma America announced the results of the Phase 3 trial. You can read more about the results here.
Want to find out more about Edaravone? Check out the resources below:
The Scientific Advisory Council (SAC) Briefing Notes
The Scientific Advisory Council for the International Alliance of ALS/MND societies consists of 9 members from USA, UK (including MND Association's Head of Research - Dr Nick Cole), Sweden, Italy, South Africa, China, Australia and Canada.
They produce briefing notes as a trusted source of information about current experimental treatments in clinical development, such as Oral Edaravone.
These notes are produced after many rounds of careful discussion between the SAC members.