The Latest Update on Translarna from PTC Therapeutics

The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and now, Translarna. The journey has been a long one.

The team at PTC are pioneers, the company learning to become a pilot while flying the Duchenne plane at 35,000 feet in a storm. The data includes hundreds of individuals who have participated in studies. Families seeing continued benefit for years now.

Update from PTC

Today, PTC’s President & CEO, Stu Peltz, sent a letter to the Duchenne community updating us on the latest news from the FDA. After appealing the Refusal to File letter PTC received from the agency in the spring, the company recently received a denial for that appeal

There are still more steps to be taken, more battles to fight to help PTC have their moment to properly present data for Translarna with a full and fair review by scientific experts at an Advisory Committee Meeting which includes the voice of the patients. But it won’t be easy.

Read the update from PTC below:

Dear Duchenne Community,

I want to update you on our efforts to bring ataluren (also known as Translarna) to your boys and young men. As you know, for more than 18 years, PTC Therapeutics has been honored to partner with the Duchenne community in our effort to develop ataluren to treat Duchenne due to nonsense mutation (nmDMD). We feel we have become part of your community and are so grateful for all of your support and dedication through the development process. All of our efforts are focused on finding treatment for those affected by Duchenne and for everyone living with a rare disease.

As you may know, earlier this year we received a Refuse to File (RTF) letter from the FDA with respect to our New Drug Application (NDA) for ataluren for the treatment of Duchenne muscular dystrophy due to a nonsense mutation in the United States. The FDA uses RTF letters to avoid unnecessary review of applications they consider incomplete. As we have discussed with you, PTC strongly disagrees with the RTF decision. The NDA is based on the results of our extensive clinical research and experience in more than 400 boys that have been treated with ataluren in our clinical trials. PTC continues to believe that the FDA can only accomplish a proper assessment of the data and analyses from our trials in the context of a full and fair review by scientific experts at an advisory committee meeting which includes the voice of the patients.

We intend to continue to seek the opportunity to have the FDA review our ataluren data and consider this therapy for approval. In August we announced that we submitted an appeal to the FDA via the formal dispute resolution process in order to elevate the discussion on the RTF to the next level of FDA management. We were recently informed that our first appeal was denied. We are frustrated and disappointed by this decision but our commitment to work with regulators and the Duchenne community to make ataluren available to nmDMD patients in the United States remains firm. We plan to elevate our appeal to the next level of FDA management, as outlined in the formal dispute resolution process. As we have said before, this is an iterative process and we anticipate that multiple cycles of appeals to progressively higher levels of the FDA may be required.

The road to develop treatments for Duchenne has not been easy or straightforward. Despite these challenges, we have persevered and worked to find a way forward. We hope that you will continue to be a part of this journey and help us by making your voices heard. Your personal experiences living with Duchenne and participating in clinical research are critical if we are to be successful in encouraging the FDA to consistently review data related to all therapies that may help those living with Duchenne Muscular Dystrophy.

Together we can continue to raise awareness about Duchenne in the effort to bring new treatment options to families as quickly as possible.


Stuart Peltz, Ph.D.
President and CEO, PTC Therapeutics, Inc.

Turning Your Experience into Data

As a community we have learned that data is critical, but equally important is what we learn over time. How individuals feel, function, and survive is not a data point, but a lived experience. Patient Focused Drug Development (PFDD) is a new science: the science of patient input. Over time we have heard anecdotal stories of benefit, of change, of the impact of Translarna. We have been and continue to capture those stories, providing this to FDA.

Our ongoing preference studies translate these stories into rigorous data. Slowing disease progression is our highest priority. It matters that individuals walk longer, move their arms longer, breathe without ventilation longer. Across the years, 99% of the individuals on Translarna continue to take the drug.

Translarna has been approved in Europe and we are hopeful this approval will be renewed. With the request for renewal, PTC will be required to conduct another study to fully understand benefit across the trajectory of Duchenne. Success in this study would ensure access for all individuals – from the very young to adult.

It is time for PTC to have the same opportunity in the US. PTC deserves a full and fair review by scientific experts at an Advisory Committee Meeting.

Approved therapies for all individuals diagnosed. This is our goal.

What You Can Do


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Comment by Andrea Cleary on October 26, 2016 at 10:17pm

Is PPMD willing to do the same for Drisapersen?

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