Anyone has any idea when Translarna is coming to the pharmacists in the EU?

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Today, I got the reply from PTC about Translarna :

"Translarna is only approved in the EU. It has not been approved for sale anywhere else in the world including the US. In the EU, access to ataluren is only through  national health systems. Because these systems are sponsored by the government, it is generally required that patients be citizens, or at least residents, of these countries.

We are currently conducting a confirmatory trial of Translarna in DMD. We expect to have results from this trial in the fall of 2015 and to apply for approval in the US, as well as full approval in the EU, in the first half of 2016. I do not know at this time when we might be seeking approval in Asia but we would be seeking marketing approval on a country by country basis, not for the entire region at once.'

We live in Vietnam - Asia, mean we cannot get Translarna. Feel much disappointed and sad. It will be late for my son.


Trinh,am in Kenya.After reading that communication to you from PTC,i am now really scared.

 I feel desperate.

Trinh thanks for sharing the response from PTC. It is very surprising that they are going to take almost 2 years to get the drug in Market. I am located in India and Translarna will not work for my son as he has deletion of exon 44, but worried that if an exon skipping drug is approved it will take 2 years for people like us to get access to it.


Rahul, since Translarna just got the conditional approval from only EU, so it will be available for citizen and residents in EU only. In USA, PTC expect they can get full approval in early 2016 ; till then patient in USA can get Translarna. After that PTC will seek marketing approval country by country. How can we wait ?

Let us hope when it gets full approval in the US it can be accessed outside the national health system,that way parents outside the US can be left with only the financial aspect to deal with and not with the issue of non-accessibility.We can only hope. 

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