But will it get approval in the U.K.?
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But will it get approval in the U.K.? -
Approval process - MNDA please comment
This is the most significant news for over 20 years.Originally posted by GemmaPob View Post
Forum monitors can you please bring to attention of appropriate Mnda management (they are almost certainly aware)
We need a formal review/plan of action etc.
One more step!
Onein400 -
Massive news.Comment
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It may well be but at $146 k a year for six months extension ?? Is it a NHS option ?Originally posted by steve001 View PostComment
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It's good news, but there has to be a solid calm approach to it.Originally posted by steve001 View Post
It's needs to be a pill really, for a small effect drug. The focus should be on developing that pill.
But it's great news for awareness month which is just coming in June!
One small step,Comment
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Hi all,
Great news indeed and now the MNDA needs to focus on how to campaign for approval in the UK. I suspect the EU will have it's own agenda on approval and whilst we are still in the "club" we don't want to start the process alone in 2 years. I and no doubt others will be raising this next month at the AGM meeting in East Midlands.
BarryI’m going to do this even if it kills me!Comment
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https://mndresearch.wordpress.com/2017/05/06/edaravone-radicava-approved-to-treat-mnd-in-usa-what-does-this-mean-for-people-with-mnd-in-the-uk
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The pertinent bit being:
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.Last edited by Ellie; 6 May 2017, 11:24.Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
Significant bulbar impairment - No functional limbs - No speech - Feeding tube - Overnight NIV - Eye gaze user.
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Just a suggestion, all those living with this illness might want to consider also registering in ALS Worldwide. The reason being there is a woman in Cambridge, England that is getting Nuedexta the medication available here in US for emotional liability that is not available in the UK, but this Cambridge woman is getting it shipped to her with the help of ALS Worldwide.
If Edaravone becomes available in the US, but with greater difficulty in the UK it may be possible that ALS Worldwide could help each individual that wants to try it obtain it.
This organization too have been monitoring the progress of Edaravone and did a write up about on their site.
xoxComment
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Hi CC,
Thanks to your pm I registered with ALS worldwide and got a same day reply. They want me to arrange a webcam discussion of 30 minutes duration. That will fun for someone who can barely make their family understand them.
Barry xxI’m going to do this even if it kills me!Comment
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A big YAY Barry!
I know they will also communicate via email, and if need be will send someone to your home.
The more I read deeper into this organization the more aware I became of how they can cross boundaries to help those around the globe effected by MND/ALS
The bottom line is they are another resource to tap into if need be, and may be able to provide additional support in areas that your NHS might not be able to cover.
Nothing to lose, and much to gain in my opinion.
Good luck Barry, keep me posted
xoxComment
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CC:
Your interest and work is much appreciated this side of the pond. Thank You.
Barry: good on you.
I would not worry too much about being understood. Those Americans would struggle to understand your accent at the best of times.
CharlesComment
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My pleasure Charles even if it’s in the smallest of ways.
Have to say we Americans absolutely adore your accents !
For some bizarre reason my daughter attempts to speak in it often. She says ‘Ello Mummy’ to me all the time, and when we go out to restaurants to eat she orders her food to the waiter in a British accent and she is very serious about it too. When I ask her after the waiter leaves ‘Why are you talking like that??’ she will say in a British accent to me ‘Talking like what Mummy??’ She cracks me up LOL
I recently watched this movie in set in your country called ‘Love Actually’ with Hugh Grant and lots of other famous British folk, it was about all different kinds of love relationships. This one character a young guy couldn’t get ‘shagged’ (as you British say) so he figured he would come to America because American girls LOVE your accents (he is so right!). So he buys a ticket to Wisconsin! LOL gets off the plane as tells the cab driver to take him to any bar, just any bar in Wisconsin. He orders a drink, and this pretty girl at the bar hears his accent, and next thing you know he is sitting at a table with her and some of her friends, and they are asking him to say random words just so they can hear his accent. It’s hilarious!! He did get shagged with the girl and all her friends! LOL This movie had a great soundtrack too loved the music in it
xoxComment
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May 06, 2017
FDA Approval of Edaravone
By: ALS Worldwide
A CAUSE FOR CELEBRATION
The anticipated FDA approval for Edaravone, also known as Radicava, has arrived a full month earlier than expected. On Friday afternoon, May 5, 2017, FDA authorization was granted to Mitsubishi Tanabe Pharma America, Inc. to manufacture and distribute this important medication that has already provided systemic benefit to many with ALS in Japan, India and to smaller patient groups, in at least 16 other countries, including the US.
At ALS Worldwide, we have had first hand experience providing compassionate use of Edaravone to a group of ALS patients since September 2016. Because of the positive trial results shared by Mitsubishi Tanabe at that time, we felt it important to enable this medication, on a compassionate use basis, to those who sought Edaravone and were able to manage the challenges Edaravone encompasses. Some US and EU patients traveled individually to Japan to bring back a several month supply for themselves. For others, we traveled abroad to import this medication for its compassionate use trial, and benefits, in the US and other countries.
We learned that the majority of those who began its use, now for as long as 12 months, have achieved benefits that outweigh some of the challenges involved in its administration. Edaravone, or Radicava, requires a PICC line or Port-A-Cath for daily intravenous (IV) injection and can take up to an hour for the daily infusion to be completed. For most, this was achieved in-home and soon became an acceptable part of their daily routine.
After 22 years since the introduction of Rilutek (generic name riluzole), we appear to have a new systemic treatment that offers important benefits to the ALS patient community. Radicava, as it is now named in the United States, is expected to be available to patients and their doctors as early as August 2017 but may be up to three months later.
In months past, ALS Worldwide has responded only to specific patient inquiries for individual compassionate usage of Edaravone, funded in whole or in part by patients and families themselves. Our role included sourcing and importing the medication, confirming its authenticity and safety, continuous monitoring of usage and helping develop local medical oversight, administration of Edaravone, and correlation of patient usage and results. ALS Worldwide has provided like services for other trial medications under the supervision of our medical and scientific advisors, to whom we are deeply indebted. This effort is part of our ongoing effort to support the ALS patient community as we have done since 2002.
What can you do now that Edaravone has been approved?
1. In order to make an informed decision about the usage of Radicava, learn what is involved in procuring a PICC line or Port-A-Cath by reviewing this document: Stanford Hospital Patient Information
2. Notify your local pharmacy that you wish to be informed as soon as Radicava becomes available
3. Contact your insurance company concerning their coverage for Radicava
4. Discuss the installation of the PICC line or Port-A-Cath with your local physician
FDA News Release
Mitsubishi Tanabe Pharma Corp news release of estimated infusion cost.
ALS Worldwide began informally in 2002 when our son Ben Byer was first diagnosed with sporadic ALS. Our efforts for Ben and eventually thousands of other patients, now in more than 125 countries around the world, has continued as a not for profit organization since 2008.
Today, we strive to help people with ALS live better and longer lives by providing compassionate care and counseling, assistance in managing ALS Symptoms, greater access to beneficial medications, information about medical devices and communication systems, caregiver support, and guidance on healthcare topics and resources.
You can learn more about ALS Worldwide through the following venue:
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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.Comment
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