Tuesday 17 July 2012

Ataluren update from PTC Therapeutics

In a recent update about ataluren, PTC Therapeutics has described the results from their phase 2b clinical trial as well as updating patients on the way forward in getting the drug to the market.

Ataluren update
Background information
Further information and links

Ataluren update

The trial, which ended in 2009, showed that ataluren was safe and well tolerated. The researchers found that a high dose of ataluren had no beneficial effects, but saw encouraging results using a lower dose. The lower dose of drug increased the distance boys could walk in six minutes and also slowed the speed at which their walking performance declined.

However, regulators in the USA were not convinced that the results of the clinical trial were sufficiently robust to allow approval of the drug at the current time and PTC is planning to start another study in an attempt to confirm the results.

In Europe, the regulatory system is slightly different and PTC Therapeutics and the European Medicines Agency are discussing whether the company may be allowed to apply for conditional approval for the drug. Conditional approval is granted to drugs whose benefits are likely to outweigh their risks, but where additional information is needed to confirm this situation. Currently, PTC Therapeutics has not set a time-scale for this process, but if the application goes ahead and is successful, further trials will be required. However, conditional approval would give patients access to the drug while the trials were ongoing.

Background information

Ataluren is an oral drug that has been developed in the USA by PTC Therapeutics to overcome a specific change in the DNA called a nonsense mutation. About 10-15% of boys with Duchenne muscular dystrophy have this type of mutation. In very rare cases, nonsense mutations occurring in certain positions in the dystrophin gene can also cause the milder symptoms of Becker muscular dystrophy.