Ataluren Phase 3 Confirmatory Trial
– Planned to commence the trial in the first half of 2014
– PTC are currently discussing the trial design with the FDA and EMA
– Planned that the trial design will be sufficient for subsequent FDA and EMA applications
Conditional EMA Application (Europe)
– Planned to submit an application for conditional approval of Ataluren
– This has been delayed until the start of 2014, due to a focus on the Duchenne Muscular Dystrophy application
What is Conditional EMA Approval?
“Conditional approval is granted based on a positive benefit/risk ratio in the available data which, while not yet comprehensive, indicate that the public health benefits of immediate availability of a medicine outweigh its risks. The company is given obligations to fulfill, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a full approval.”
I found the following two graphs in a French presentation recently. They show the changes in FEV1 during the previous phase 3 Ataluren trial.
The first graph shows that the Ataluren group dropped -2.5% (relative) from their baseline, but were 3.0% higher than the placebo group.
This is the first time I have seen these graphs. They show that those taking Ataluren without inhaled antibiotics were stable on average (-0.2% relative). The other three groups (two placebo and Ataluren with inhaled antibiotics) were not stable and decreased between -4.4% to -6.9%. The stability seen with Ataluren is encouraging; if people with CF are stable and not declining a few percent per year, this is very significant.