Radicava (edaravone) approved by the FDA in the US

[Ellie]

In the absence of a cure, I reckon people would be extremely happy to slow down their progression, especially as Edaravone worked best in the newly diagnosed (according to the trial data)

We'll see when it's available in the U.S. this autumn.

[steve001]

Thank you for your enquiry to Mitsubishi Tanabe Pharma Europe concerning Radicava(r) (edaravone).

As you state Radicava(r) has now as of Friday been approved in the USA by the FDA for use in the ALS indication. However it is envisaged that it will be about three months before everything is in place to market the product in the USA. Currently edaravone has only been approved and marketed for sale in Japan and South Korea. The Mitsubishi Tanabe product is not current available in any other countries and there is no named patient supply/patient support program for edaravone nor any active clinical studies in ALS patients. Mitsubishi Tanabe Pharma is currently not able to supply the product anywhere outside Japan and South Korea and then only through the organised healthcare distribution network.

Mitsubishi Tanabe Pharma is try to make this drug available in other regions. However at the moment, no decisions have been made.

I am sorry not to be able to provide you with additional information at present, but thank you again for your enquiry.

Kind regards

Mitsubishi Tanabe Pharma Europe

[Barry52]

Thanks for posting this Steve. Not an unexpected reply but at least you have cleared it up for others.

Barry

[Onein300]

Hi - I'm coming up to 3 years since this disease started. This drug slows the progress down for an estimated 6 months. At which stage do you say that I don't want it to slow down? I want a cure rather than something to slow it down. I cannot get excited about this news.

I agree. Actually the trial showed a reduction in decline from (on average) 7.5 alsfrs points to 5.01 points over the 6 months of the trial. Everyone declined by 5.01 points, which as we all know is significant disability. It does not relate or map to 6 month delay. It cannot be extrapolated.

Now with the highly subjective nature of some alsfrs points, this as you say is really not that exciting.

Also it does appear to be in first year or months from diagnosis (although I haven't seen the detail here). Now not wishing to be more difficult, but we all know that the wide variation in this disease, especially in Year 1 or 2 when our bodies can actually adapt better than imagined and dare I suggest that our alsfrs scores can go up and down?

But the press/awareness that we can get will be great.

[ccinjersey]

True not the cure, but still a reason to be positive because its something that worked and shows some progress is being made, and where this worked something even better can and will come as scientists, and researchers are encouraged to push harder. Who knows maybe when more people have access to it there will be more positive reports about the effects of it ...

Even small step forward is better then no step forward, even though its not enough for so many that need much more, and need it now.

xox

[John14]

I agree this seems to offer modest effects at slowing down the progression of the disease and comes 20 years after Riluzole which has a similar effect, we are still some way off a cure but it does look as though this may be a step in the right direction. John

[Onein300]

I agree this seems to offer modest effects at slowing down the progression of the disease and comes 20 years after Riluzole which has a similar effect, we are still some way off a cure but it does look as though this may be a step in the right direction. John

It is a step in the right direction.

Consider this..

Because of the MND disease measuring issues (ie subjective alsfrs) typically we have to run all trials with fast progressing cases. Unless the disease modifying is dramatic you just cannot see it in slow cases in a 6 to 12 month trial.

Now if a drug shows effect on fast cases, who knows what it might do with slow or very slow cases? Depending on mechanism it could actually be significant!

Riluzole, for example, has had no trials greater than 18 months. People have been taking it for many many years in some cases. So the 2/3 month life extension could be dramatically out for slower cases.

[njm]

Onein400

Spot on. Very good analysis of the potential of Riluzole if taken early or in slow progression.

Charles

[Ellie]

But progression is not linear... Not in ALS anyway. And therein lies a significant issue in the measurement of any slowdown; it is impossible to know how anyone's ALS will/would have progressed anyway.

I was deemed to have relatively fast progression for my first 12 months, losing more than half of the possible 48 points on the ALSFRS-R scale 12 months after diagnosis (Definite ALS diagnosis) I lost 9pts during my first 6mos and 16pts during my second 6mos.

Year 1 - lost 25 points.
Years 2 - 10 - lost 14 points in total.
Current ALSFRS-R score is 9 points.

Ellie.

[njm]

Thanks Ellie:

The voice of experience and reason.
We (I) are clutching at straws trying to predict and thus gain a modicum of control over this disease. But truth be told it has a whimsical mind of its own.

Charles

[Just Me]

While researching Edaravone (Radicava) I ran across the following link that does a side-by-side comparison between it and Riluzole and something called are RCH4. First, I would love any thoughts with regard to this comparison. Additionally, any information about RCH4 would be very helpful. http://www.als-new-drug.com/riluzole-edaravone-rch4

[ccinjersey]

Hi Just Me

Who is providing the RCH4 drug for compassion use ?? Has there been studies done on it, and what were the results ?

CCxx

[Just Me]

Hi Just Me

Who is providing the RCH4 drug for compassion use ?? Has there been studies done on it, and what were the results ?

CCxx

Those are great questions. I would really like to know. That is one of the reasons I posted this here. I'm hoping someone on the forum might have that information.

Michael

[Just Me]

I did a little more research about this. And, I ran across this link. It's on a website called patientslikeme.

[njm]

A cursory look at this and several things stand out:
1. There are minimal, if at all, side-effects.
2. All report significant benefits.
3. It is very easy to take.
4. It is not expensive (this point confuses me. Is this because it is a subsidized trial?)

Given this info I would be very keen to get onto this. cost would be the prohibitive factor.
It appears that it slows progression down.

Charles