The FDA and Ataluren (Translarna)
I am not seeing any posts or comments about the devastating news that the FDA has again chosen to sabotage Ataluren, by issuing a "Refusal to File" letter to PTC, which then led to the company announcing that it is laying off 18% of its workforce in the US, plus a huge fall of the price of its shares. The news is more than one month old, and yet I only learned about it by chance, by checking a financial news website.
In the past I submitted many comments about this key drug and the idiotic way in which the FDA has treated this very promising (so far unique) treatment for nonsense mutation DMD,which HAS DEFINITELY SHOWN A MARKED IMPROVEMENT FOR PATIENTS AT LOW DOSES OVER THOSE ON PLACEBO.
In Europe, PTC was granted conditional approval more than 15 months ago. Many patients and parents around the world were eagerly waiting for the FDA to follow the same path.
What is this? Some kind of jingoistic "war" between the FDA and the EMA?. Are the Americans trying to make the point that they are reeeaaaaally careful and responsible, while the Europeans are a bunch of happy-go-lucky bums?
I've been following the story of PTC124 since early 2003 (THIRTEEN YEARS!!) My son is now almost 26 years old. Everybody knows that Ataluren WORKS. There is a 47 meter difference in the 6MWT in favor of Ataluren treated boys against those on placebo. I've had enough, really.
Replies to This Discussion
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Permalink Reply by Michelle Barshay on
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I've been wondering too why people are not up in arms over this. Our family and friends are devastated. We've been waiting for this drug for our son, Connor. We were not part of the trial and therefore don't have access to the drug. Connor is turning 9 in a few months. This is the right time to begin atalauren. I thought the FDA understood our community and where we stand. I thought they understood that a drug that is somewhat effective is better than nothing at all. All three drugs that are up for approval will benefit our boys greatly. Why can't they just approve them?
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Permalink Reply by Shelly on
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Even if it benefits mildly, would be a great support to the boys. FDA should understand!!
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Thank you very much for your posts, Shelly and Michelle. I see now that I am not the only one who is angry about such and negligent conduct by the FDA. Anyone from the staff of PPMD cares to comment/explain?
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Permalink Reply by Jay Griffin on
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I share the same feelings... It's totally frustrating...
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Hey Bernardo our pain is same, all of us in the same boat. Though Ataluren can't work on my son, he needs exon 44 skipping as he is missing exon 45 but I can very well understand how we feel when there is a drug which can benefit your son , not available. Try to reach out to Europe and get the medicine if possible. Good luck!!!!!
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Thank you very much Shelly. We were about to try getting it from Europe. I hope the FDA decision doesn't trigger a panicky reaction from the Europeans. I also very much hope that the exon skipping drugs move forward so that your son and other boys can benefit. Good luck to you!
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Permalink Reply by Andrea Cleary on
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The Drisapersen refusal by the FDA is a similar story.
If you've seen the videos of my son Simon 15.5, you know it works! (And Gavin Carlone 10, Maxime from Belgium and others)
Lets work together to get the FDA to understand their stance is unacceptable!
They are taking written testimony right now (until April 11th deadline) for the Eteplirsen Advisory Committee, so write the FDA and let them know that we want and need ALL of these drugs approved NOW!
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Andrea, where is the video
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True!!! Let's work out... Write to FDA .. Andrea can you please give me the email id where we can write! Actually I am in Australia so tell me how can I participate.
Thanks
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Time to wake up. Our whole community should unite to save our sons. These drugs, even if making 0.1 percent, can increase our kids mobility and eventually their life span. FDA!!!! Please approve the drugs,they are life saving
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Permalink Reply by Rahul Deshpande on
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Ataluren is approved in Europe and available in many European countries. Its proven beyond doubt that it benefits children with non sense mutation. What more does FDA want. Why do they want more data I don't understand. The sad story of Drisapersen is known to all. I have seen presentation and videos from parents telling that Drisapersen is helping their children. Also no reported serious safety issue. Still it was rejected. What more does FDA want. All the pressure exerted by the community has not helped so far. On the other hand we have seen many drugs being advertised which have benefit in only x percent cases and have a long list of side effects, still those drugs are approved by FDA. For the DMD drugs at least the FDA can give a conditional approval where they state that it has limited benefit and has some side effects. That will then make those drugs available and parents and patients will have the option to use them or not.
Now all of us are eagerly awaiting the fate of Eteplirsen.
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Jay, You can see the videos of my son, a 15.75 yr old Drisapersen participant, on my Facebook page at https://www.facebook.com/andrea.cleary.52 Please scroll down to March 10th and 11th
Jay Griffin said:Andrea, where is the video
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