Fortbiken against Duchenne - 6 uren van het Fort van Borsbeek

Fortbiken against Duchenne - 6 uren van het Fort van Borsbeek
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Deze mountainbiketocht is een initiatief van het Nederlandse Duchenne Parent Project en wordt georganiseerd in samenwerking met de Nederlandse stichting "Evenementen voor Maatschappelijke Organisaties Foundation (Emolife)".

donderdag 6 december 2012

PTC Therapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Ataluren in Duchenne Muscular Dystrophy



Dear Friends,


As announced this morning, PTC is very pleased to have received the acceptance for review of our application for conditional approval in the EU. This is a historic moment for the Duchenne muscular dystrophy community and for PTC, as this is the first filing of a marketing authorization application for a new chemical entity for DMD. 


We would like to thank all the patients and families who have participated in our trials and have help guide our efforts over the last decade. The results of these clinical studies not only served as the foundation of the MAA, but provided PTC and the DMD community with valuable insights into the natural history of Duchenne muscular dystrophy and the potential impact that ataluren may have on disease progression.


In terms of our corporate strategy and next steps, PTC is moving forward not only with the EU MAA filing acceptance, but also with increased U.S. regulatory clarity. Based on our conversations with the FDA and EMA, we plan to enroll the first patients in our Phase 3 confirmatory trial as early as the first quarter of 2013. We have developed the design of this study in collaboration with our DMD investigators and patient organizations and with input from EMA and FDA – for which we are extremely grateful. The results of this confirmatory Phase 3 study will hopefully serve as the basis for full approval in the US and EU.


We expect to have additional information for patients and families on our website, and in January. Parent Project Muscular Dystrophy is planning a webinar at 12 noon EST on January 17 so that we can provide additional information on the trial. PPMD will have details about the webinar as well as how to submit questions in advance on its website,, within the week.


As you know, the path to approval for a treatment in a disorder that has never had a drug approval is not easy or straight-forward, but our corporate vision and sense of urgency have never wavered – even when faced with challenges. Instead, our energy and enthusiasm to advance ataluren as rapidly as possible continues to grow, and is reinforced by our robust foundation of preclinical and clinical data. We are supported by our collaborators and investors and are inspired by the courage of these amazing boys and young men, the dedication of their families and the commitment of clinicians, researchers, physical therapists, site coordinators, patient advocates and all members of DMD community.


I want to take a moment to thank the team at PTC, which has been relentless in advancing ataluren from the initial idea through all the multiple steps leading to this milestone. Each team member has worked tirelessly and shares a deep commitment to our goal of transforming the lives of patients.


Again, we thank you for all of your efforts and support! 





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