Outcome
Treatment with Edaravone has showed some signs of beneficial effect in clinical trials. Other trials are underway, which are looking to see the impact Edaravone has on markers of the disease within the body (biomarkers). You can read more here.
Both Oral and Intravenous Edaravone (Radicava) have been approved as a treatment of ALS in the United States and a select few other countries. Radicava was reviewed by the European Medicines Agency (EMA) but Mitsubishi Tanabe Pharma America withdrew the application. At the time of the withdrawal, the Committee for Medicinal Products for Human Use, of the EMA, was trending towards not approving Radicava. You can read some FAQs about the withdrawal here.
A different formulation of Oral Edaravone was tested in a clinical trial across Europe. This trial did not meet its primary endpoint, suggesting it was not beneficial for people with MND. You can find out more here.
What is the drug thought to do in the body?
Edaravone was originally developed to treat acute ischemic stroke. 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, but in MND the metabolism can be heightened and increase the impact of oxidative stress. This can result in cellular damage resulting in excessive accumulation of free radicals. Edaravone acts as a ‘free radical scavenger’ helping to reduce the effects of oxidative stress, with the aim of slowing the progression of MND.
Two different formulations of edaravone have been made, including an intravenous (straight into the bloodstream) form and an oral form.
Previous Clinical Trials
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 revealed that after 24-week participants 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.
Phase 3
The Phase 3 clinical trial tested an oral suspension formulation of edaravone (MT-1186), but was paused in March 2020 due to the COVID-19 pandemic. The trial recruited 185 participants. The trial showed that it was safe and well tolerated and 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.
Phase 3b
The Phase 3b trial investigated and compared the efficacy of two dosing reimens of oral edaravone (MT-1186) in people with ALS. The trial used the change in ALS Functional Rating Scale-Revised (ALSFRS-R) score from baseline. The trial aimed to see whether taking a daily dose of oral edaravone would be more beneficial for people with MND compared to the FDA approved on/off 28-day cycle dose. Pre-planned interim analysis from the trial showed that if the trial continued to the end of 48 weeks there would be a low chance of the daily dose being more beneficial than the on/off 28-day cycle dose. This led to the trial ending early. You can read more about the trial here.
Latest News
2023
August 2023 - The Phase 3b trial comparing two different dosing regimes has been stopped early. Pre-planned interim analysis suggested that there was a low chance that the daily dose would be more beneficial than the on/off 28-day cycle dose at the end of the trial. You can read more here.
2021
Dec 2021 - Mitsubishi Tanabe Pharma America announced the results of the Phase 3 trial. You can read more here.
Resources
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.
You can find the briefing note for Oral Edaravone here.
Last updated: 21/08/2024