On Friday 5 May in America, the FDA, the organisation that approves drugs, announced that they’d granted a licence for the drug known as a Edaravone (to be marketed as Radicava ) for the treatment of MND. It’s unexpected news and we’re currently working out what this means for people with MND in the UK. Below is more information on what we know so far:
What is this drug and what does it do?
In clinical trials, Edaravone has been shown to slow the progression of MND potentially helping people preserve function longer. Some of the clinical trial results have shown that Edaravone only works on a subset of people at the early stages of the disease – we are seeking to confirm this.
Edaravone is an antioxidant drug that works by mopping up ‘free radicals’ in the body. Our cells have quite effective ways of dealing with free radicals, but these ‘cellular defences’ become less and less efficient with age.
As we age, our energy production processes lose efficiency, causing a ‘double-whammy’ of not only more free radicals being produced, but also less effective ways of dealing with them. When neurones are damaged, as happens with neurodegenerative diseases, then everything gets exacerbated even further, leading to a vicious cycle of events.
It’s a bit like sparks escaping from a campfire – if there are too many sparks and you don’t keep an eye on things, you could end up with the forest ablaze. There’s more information on earlier post on our research blog.
How would people take Edaravone?
Edaravone is administered intra-venously (IV). People with MND would receive the drug every day for two weeks, then take a break for two weeks. A company called Treeway are currently developing an oral preparation of the drug.
What is the process for licencing this in Europe?
We are in contact with Mitsibushi-Tanabe in the USA and have asked them to connect us with their European office in order to understand their plans for licensing in Europe.
The company will have to apply to the European Medicines Evaluation Agency. The drug has already been registered with EMEA as an orphan disease candidate, which means that any licensing application will be fast-tracked. EMEA approval, however, does not ensure UK approval and the drug would need to be approved by the Medicines and Healthcare Regulatory Agency. New medicines are usually also reviewed by the National Institute for health and Care Excellence, which makes recommendations on the cost-effectiveness to the NHS. There is a process for joint MHRA-NICE review which the company will doubtless pursue.
The licencing process does take time, so the company could also apply through the Government’s Early Access to Medicines scheme, which aims to make a drug available where marketing authorisation is not yet approved and there is a clear unmet medical need.
Where can I find out more?
More information on Edaravone is available on the ALS Association website. More information on clinical trials in general is available on our website our website and in our research information sheet. As we learn more about the developments of the drug we will keep everyone updated.