Wednesday 30 November 2011
Ataluren extension trial for Duchenne about to start
PTC Therapeutics has affirmed their commitment to develop Ataluren to treat boys with Duchenne muscular dystrophy and announced progress in starting an extension trial in the UK, Europe, Israel and Australia.
The Muscular Dystrophy Campaign is keeping a close relationship with the pharmaceutical industry to get first hand information about ongoing clinical trials. We recently met with Diane Goetz, Director of Patient and Professional Advocacy at PTC Therapeutics to discuss the long awaited ataluren extension trial for boys with Duchenne muscular dystrophy. The initial results of the international phase 2b clinical trial showed that the lower of the two doses tested was more beneficial than the higher dose and because of this unexpected outcome all ongoing studies had been halted in April 2010. In the US all boys who participated in the phase 2b study have been receiving the lower dose in an extension trial since November 2010 but getting an extension trial started in Europe has been slow.
Diane reassured us that PTC Therapeutics remains committed to continue with the trial here in the UK as quickly as possible and has been working with leading clinicians and the regulatory bodies to ensure that all the boys who participated in the phase 2b study here will be able to gain access to the drug. The company has now made an announcement that it aims to submit the necessary paperwork to the regulating committees in the UK, Europe, Israel and Australia by the end of December 2011. When the extension trial will begin will depend on how long the various committees in each country take to process the paperwork and give approval for the extension trial to start.
Dr Marita Pohlschmidt, Director of Research at the Muscular Dystrophy Campaign said:
This is finally good news for our families who have been waiting patiently for a positive decision. The Muscular Dystrophy Campaign will continue to work closely with PTC Therapeutic to ensure that the voices of our families are heard and we will push for the development of Ataluren to stay on track.
Background information
Ataluren is an oral drug that has been developed in the US 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.
The full results of a large international phase 2b clinical trial of ataluren were announced in October 2010, read about the results here.
Further information and links
Read the update circulated by PTC Therapeutics.
More information about Duchenne muscular dystrophy.
Read about the Duchenne research we fund.
If you have a question about this or any other research, please email us: research@muscular-dystrophy.org.
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