- The first treatment for the underlying cause of Duchenne muscular dystrophy -

SOUTH PLAINFIELD, NJ – May 23, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

"We are very pleased with the outcome of the CHMP review of Translarna's marketing authorization application (MAA) and the level of engagement we experienced with CHMP members throughout the review process," said Robert J. Spiegel, M.D., Chief Medical Officer of PTC Therapeutics, Inc. "We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. It is important to note that this journey continues through the completion of our Phase 3 Translarna confirmatory trial in nmDMD (ACT DMD) which is a high priority for PTC and the DMD community."

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But that takes courage to design a trial that is'wholistic'. Almost everyone chooses the easy way -6MW test. Look at the Taldafil trial for example..what a cop out!. What drug sponsor wants to find out from the regulators at the phase 2 that their trial design was "too innovative'?
until the regulators open the door (with plenty of pushing from us) it just won't happen

We must hope there is more to come,if not from PTC then from any of the other researchers and drug developers.But as things stand,its still very good news that some boys will now have a chance to live.It is only that ataluren for ambulatory boys kind of got me confused.

I might be wrong, but the 6MWT is the main recognised marker by the FDA. As such anyone in trials will need boys who are ambulatory in order to demonstrate effectiveness. For non ambulatory boys they will either need to get it outside of the trial on compassionate grounds or wait till it's approved (or not)when they will no doubt extend it to all  boys,regardless. So in short,it's because they want to get regulatory approval that the 6MWT remains.  It doesn't mean this, or the other treatments , won't work for younger/older boys.  I guess it will take the community to push for 'off label' use on compassionate grounds.

There are other markers, such as heart/lung strength and a number of bio markers (such as CK and the work done by a number of the companies). If they accepted a broader sweep from non ambulatory boys it would open these trials to older boys.  

Hello Steven

I think your point explains it.With the 6MWT being the marker,they have to work with ambulatory boys.Thanks.

That's why we, the patient community,  have to hit home the point that we need different biomarkers and end-points, not just a one fits all 6MWT.

The EMA, FDA, Health Canada are starting to get the message that in the rare  disease arena, especially progressively fatal disorders, we cannot wait and rely one single end-point (black and white), we have to be given the drugs so we can help confirm  effectiveness using various markers. We need some grey in the bigger picture.


Perhaps a bad choice of words on my part - but I agree totally that our advocacy has to start early for various objective measures etc   I think innovation in trial design would have the reverse effect - there would be less grey areas about efficacy.  Our kids are living longer with better care, medications, therapy and early steroid intervention and in 5 years time we can expect the trend to continue so we need to know with certainty that stabilization is due to the innovative therapy.

Right, we need access to the drug to be able to get to that point. I'm thinking along the lines of "after-market" seveillance, or on-going confirmatory studies. Of course no one wants to be taking a non-effective medecine, or one that shows up with safety concerns years down the road.

My concern now is that the EMA will follow the CHMP's decision, and not give approval for the boys not walking. Then we have to wait for  3 more years. Our boys will be weaker, and perhaps for some - too late. We have to have the parents organisations to try to convince EMA to change the decision now. 

Next step will be the FDA, and they will most likely follow EMA's decision and not give acess to non ambulatory boys.

This is unethical and cannot be allowed. What's the difference in the muscles to a boys who's not walking and the ones who walk ? Nothing. Same disease - same progression. Every boy with a nonsense mutation will be helped with this drug. Some will be helped more - those who are younger. The older boys might keep their upper body strenght for longer. And it can also help heart and lungs. 

I was hoping for a comment from PP too. 

I'm absolutely floored by this possibility. If these treatments tested on 6MW test are only available to ambulatory patients then I may as will start planning the funeral.

David, no, it's just till they get approval. Trust me all the companies in this space want to offer the treatment to as wide a community as the regulator will allow.  Treatments are closer now than they have been for years. Be hopeful.

We have to change the way trials are set up. Right now, if they have to prove there is an improvement of say 30 meters on a 6MWT to get a drug approved, then of course they cannot let non-ambulatory kids in. We need to convince them that we won't accept these trial designs anymore, that there is more to life than just walking (lung function, upper body strength, energy level, less depression, caregiver burden, whatever). Our only alternative, as David mentions, is a non-alternative (to do nothing). Not acceptable when these new treatments exist NOW and more coming soon.

Why would CHMP specially mention an age minimum and ambulatory status then? why would any approval be limited in this manner?

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