EMA grants Conditional Approval for Ataluren


Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Duchenne, a landmark decision and a sign of hope for all. While Ataluren is targeted at a small subset of patients (13% +/-), the conditional approval is a sign that the EMA worked very hard to understand Duchenne, that they recognize this unmet need, the progressive and debilitating process of Duchenne and the urgent need to treat.     


I recognize it is a bittersweet moment for every family whose son, grandson, cousin, friend does not fit the criteria for Ataluren, as every person diagnosed needs and deserves access to treatments. The amazing and wonderful news is that once one drug is approved, there is a path forward for all, a door has opened, a regulatory agency intimately familiar with weighing benefit and risk, safety and efficacy, a regulatory agency that recognizes need and urgency, and agrees to move forward.   


But the first approval is, well a FIRST, a sign of change, a gateway to understanding and recognition that regulatory agencies understand that rare disease must necessarily be viewed through a different lens than common disease, that the patient voice and their view of benefit and risk is integral to the process, that the progression of the disease is well known and from the day DUCHENNE is diagnosed, there is little uncertainty about progression. It is well  known and is on replay in the minds and hearts of everyone who has ever known and loved an individual with the diagnosis.  


Conditional approval is used by the EMA. It enables individuals who fit the criteria to access the compound and ensures reimbursement. It also requires that the sponsor (PTC Therapeutics) complete the Phase III study, submit the full application to the EMA for analysis and a final decision for full approval.

Conditional or Adaptive licensing is not currently used in the US. In our white paper (Putting Patient’s First…) we recommended adaptive licensing/conditional approval, suggesting that once safety is established and a trend toward benefit, conditional approval is an appropriate vehicle, recognizes the urgent needs and provides access.     


While the label for the Conditional Approval (5+ ambulatory) is narrow, PTC plans to conduct studies in young children, as well as, the non-ambulant population.  PTC recognizes that ALL individuals with a nonsense mutation need and deserve access to therapies and is working with UPPMD to organize a webinar with the next 10 days. They will describe their plans for moving forward  with the FDA and answer questions you may have.  


Take just one moment and celebrate today. This is a very good day for all of us, but our work is not complete. We have a pipeline full of hope and need to continue to expand our robust pipeline. The MD-CARE ACT has played a major role and needs to continue to serve this community. The Draft Guidance is nearing completion and will be submitted to the FDA.  Members of the Steering Committee and Working Groups have suggested the Guidance should be submitted to EMA, as well, in an effort to promote harmonization in the US and Europe.  And together we have worked to raise awareness, encourage regulatory agencies to understand this urgent need. 


And today, the EMA’s decision is a good sign – Strength Happens Together.

Read PTC's News Release

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Comment by Ricardo Rojas Caballero on June 17, 2014 at 10:17pm

Beside is very very great news, there some legal way to DMD boys outside Europe can access to the drug? Or only Europe citizens can?

To many DMD families have this question

Comment by Wendy on June 16, 2014 at 4:59am

I am a mother of DMD boy in China,  happy with tears when saw this great news.

Though we now have less hope to have this drug, we still wish the boys that have NM-DMD in world wide could have this drug soon, as they can't wait for too long time..

Comment by Trinh Nguyen on May 24, 2014 at 9:18am

My son is on phase III trial. I think he's placebo case since I see his decline. I do pray so, then I still have hope. Shall we get the drug in the next 3 months ?

Comment by Peter on May 24, 2014 at 5:59am

I have read the news and shed tears I hope it will change the life of my son.

Comment by Robin Taylor on May 23, 2014 at 2:35pm

To everyone at PPMD and PTC Therapeutics,

Thanks for the update and working so hard on the behalf of all boys with DMD and their families.  You keep our hope alive.

Agree with David --- check out where your local representatives stand on the MD-CARE ACT.

Wonderful day for the DMD Community,

Robin (Louie's grandma)

No Bad Days.

Comment by David on May 23, 2014 at 2:29pm
DMd parents and family members take note! Elections matter! Find out if you're local rep supports MD-CARE ACT and if they don't change your vote

What a terrific day

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