November 25, 2013

November Ataluren Update

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.

Slide 17 Ataluren

The first graph shows that the Ataluren group dropped -2.5% (relative) from their baseline, but were 3.0% higher than the placebo group.

Slide 18 Ataluren

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. 

Sources: PTC Press Release, Nov PTC Investor Conference, Press Release that explains Conditional Approval and French PowerPoint Presentation 

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Join the conversation! 3 Comments

  1. First new data for a while on this, thanks!

    Reply
  2. do you know where I could find a graph that shows the change with kalydeco?

    Reply

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1. Class 1 Nonsense Mutations, 3. PTC - Ataluren