- 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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This is great news! It means that PTC's application for conditional marketing authorization will now go ahead. They still have to get that conditional approval from the EMA, but it is certainly a huge relieve to see that things are moving on.


They mentioned "The CHMP opinion will form the basis for a European Commission (EC) decision as to whether to formally grant the conditional marketing authorization. The European Commission will review the positive opinion from the CHMP and generally delivers its final decision within three months. The conditional marketing authorization would authorize the company to market Translarna with unified labeling in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway."

So, we will wait for another 3 months to have Ataluren ?

Yes Trinh, approximately three months to go for conditional approval. I am afraid there has been some confusion in the way this has been announced, because authorization has not been granted yet. However, the positive opinion issued by the Commission (CHMP) means that the conditional approval is practically assured now. I really hope this time we will get there. We need to understand exactly how this will work once PTC gets conditional approval within the European Union. What patients will be allowed to purchase the drug, how much will it cost, etc. I look forward to the webinar that they are supposed to announce soon.  

Dear madam/Sir

I am also very pleased for approving atalurenm but I would like to know when I want to get from market.



I hate it when articles state "only 10-15%" of DMD patients can benefit. It's thousands of people world wide! That simple.

Hello all

I think this is good news too, BUT once again the non ambolatory boys are forgotten ! The CHMP's members will recommend that EMA (european medicines agency) will give approval to boys from the age of 5 and AMBULATORY. 

Every boy not walking, and not in an extension study, will not receive Ataluren. 

For our boy, no drug. He has the same disease - same progression. Same mutation. What is this ?

For the boys who are no longer walking, upper body strength is crucial. Ataluren had no serious side-effects. That's why I cannot understand why this is happening. 

Would love to have a comment from PP. What should we do here in Europe ? Remember, this can also happen when FDA are going to give approval. They will most likely look to the EMA. 

Anyone know if the webinar with PTC has been published ? I think this was one of the subjects, and would like to see PTC's answer. 


Dear David

I hope your day is well with your loved ones.I am not much of a science person so i find most of the scientific content to do with DMD research abit intimidating.However,i am just curious about the fact that Ataluren is likely to be approved for use in boys from the age of 5 and ambulatory only.My son has been non-ambulatory for almost three years now and his upper body strength is not doing that great.Would it then mean that this therapy may not have any positive effect to non-ambulatory boys who still need to retain their upper body strength uncompromised?Have a blessed day.

I've never heard of a drug approved for use with ambulatory conditions attached. That to me is absolutely immoral.

I know of no scientific reason why Ataluren would not benefit non ambulatory boys that suffer from nonsense mutation
I think perhaps the EMA may need some of the same intense education from patient advocates as FDA has received. The idea that any DMD treatment would be approved like that is patently absurd and unscientific.
I will keep running races and organizing fund raising events with the expectation that our friends at PPMD will take on that task.

Perhaps they have less data on non-ambulatory and intend to confirm the observable benefit to ambulatory ongoing, which is another reason patients should have early input about outcome measures and trial design. In hindsight, holistic and objective measures that give power to the results, should be built in from the start especially taking natural history into account. I think they mentioned they are working on expanding applications.

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