- 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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Steven, my understanding - and happy to be corrected - is that the approval is conditional on them doing further trials. As the main marker is the 6MWT, then the boys have to be ambulatory. If if gets full approval, then it's likely to be offered to all. I would expect that to also be conditional on some form of marker(respiratory strength?) that demonstrates it works in younger and older boys.

This is my understanding also. This is a recommendation for an early approval, and it's based on the Dmd population that was included in the trials thus far.
Ok so we are saying that conditions are placed only on some temporary period called 'early approval' and some time later under 'full approval' then conditions are removed?

Still frustrating but certainly better than what I *thought* you were saying.

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