The Jett Foundation Fighting Duchenne Muscular Dystrophy

Open Letter to PTC Therapeutic’s CEO, Dr. Stuart Peltz

February 24, 2016

Stuart Peltz, Ph.D.
Chief Executive Officer
PTC Therapeutics Inc.
100 Corporate Court
South Plainfield, NJ 07080-2449

Dear Dr. Peltz,

Yesterday, the Duchenne community learned that translarna, an experimental drug manufactured by PTC Therapeutics that aims to restore dystrophin in muscles for nonsense mutations in Duchenne, received a Refuse to File letter from the Food and Drug Administration (FDA). It is our understanding that PTC Therapeutics completed a rolling New Drug Application on January 8, 2016.

Our first consideration is with the patients currently on translarna. We believe it is important to communicate PTC’s outlook on the path forward and commitment to the Duchenne program.

Importantly, the Duchenne community prides itself on being informed advocates. We understand there can be many reasons for a Refuse to File letter. In the spirit of transparency, we are hopeful you will provide the necessary information needed for the community to understand the circumstances that led to the FDA’s actions. The Duchenne community’s ability to advocate for safe and potentially efficacious drugs hinges on open and consistent communication from the sponsors.

On behalf of the patients and families who are affected by these recent actions, thank you for your consideration and timely response to these requests. We look forward to the additional clarity.

In partnership,

Christine McSherry

Jett Foundation

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