A note from Debra

Thoughts about PTC

Posted on July 25, 2016 5:32 pm

The announcement that PTC recently submitted an appeal to elevate the “Refusal to File” of Translarna (also known as ataluren and PTC124) to the next level of FDA management is an opportunity for the Duchenne community to raise their voices and let their experiences and opinions be heard!

Translarna is the only therapy under investigation in the US for boys with Duchenne caused by a nonsense mutation.  It has been studied in hundreds of boys, has a favorable safety profile and is currently approved in Europe. Children in over 20 countries, except for the United States, are receiving therapy outside of clinical trials.  We would like to see the FDA accept this application and review Translarna, just as Europe has!

We can’t afford to lose another generation of boys!  Act now and let the FDA and other decision makers know about what slowing of progression means to you, family members and loved ones.  Tell them about your experiences on Translarna and your hope for access to this therapy.  Share your stories through social media, call your local reporters, meet with your congressional representatives and write to the FDA – they say they want to hear from patients!

We need to act fast and support this application.  Every day that this is delayed is a loss of muscle and function!

What is the formal dispute resolution process?

The formal dispute resolution process (FDR) was created by FDA to provide a means for resolving disagreements between FDA reviewers and a company that has submitted an application for a new drug. Because a wide variety of complex and important scientific or medical issues may come up in the review process, FDA designed the FDR to allow senior FDA management to engage in an open, prompt discussion of disputes between the sponsor and the review division. There is a draft guidance document from FDA that establishes the framework for the FDR process, and which sets specific timeline goals for appeals. If sponsors are not satisfied with a decision after appeal, they may appeal again to the next level of FDA management, potentially all the way up to the Commissioner of the FDA. FDA officials have publicly suggested that the FDR process is the appropriate regulatory path for sponsors when disagreements about refusal-to-file decisions arise.

Summer Road Trip

Posted on July 13, 20164:49 pm

  Guest blog by Hawken Miller Many of the things I don’t think are possible for me to do really are. Once I get over my initial apprehension and thoughts about what I won’t be able to do and I open my mind I find that anything I set my mind to is possible. This Read more »

BioMarin Update for the Duchenne Community: 7th July, 2016

Posted on July 7, 20163:00 pm

  BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053. Update for Patients Receiving Drisapersen, BMN 044, 045 or 053 in Clinical Read more »