Enough is Enough

Duchenne families to the FDA: Enough is enough. No more regulatory delay. It’s past time to say yes to safe and effective treatments for Duchenne Muscular Dystrophy

The Problem

Duchenne Muscular Dystrophy is an always fatal disease for which there is no currently available treatment. It affects about 1/3500 boys born every day. Boys are diagnosed between the age of 2 and 5; they will stop walking before becoming teenagers and many will die before turning 21.

For over three years, a group of 12 boys have been enrolled in a clinical trial of eteplirsen, an experimental therapy being developed by Sarepta Therapeutics of Cambridge, Massachusetts.

During that trial, the drug has proven to be 100% safe; muscle biopsies have confirmed that all of the boys are now producing dystrophin, the missing protein that leads to Duchenne; the study has consistently demonstrated dramatic improvement in mobility for boys on eteplirsen when compared to those who are not being treated.

Despite these very positive results, the FDA has not yet allowed the company to file a New Drug Approval (NDA), the first formal step in the FDA approval process.

The FDA has failed to provide consistent guidance to the company.

The FDA continues to inexplicably add requirements and delay the process… while boys with Duchenne continue to suffer, getting worse every day.

What We Need

FDA Accountability | Over the past two years, the FDA has twice changed course, further delaying approval of this safe and effective treatment. The FDA must not change course again. The FDA must be consistent and Congress must hold them accountable.

Enforce FDASIA | As House and Senate committees consider new FDA reform legislation designed to promote early access to treatments especially for rare diseases, Congress must exercise its oversight responsibilities to ensure the FDA is following congressional intent and utilizing the tools Congress provided in the FDA Safety and Innovation Act (FDASIA).

NDA Filing and Rapid Approval | Sarepta is planning to file a New Drug Application in mid‐2015. The FDA must commit to: 1) allowing the NDA to move forward without any further delay and no new requirements for additional data; 2) earliest possible consideration of the NDA; and 3) based on the evidence compiled during more than three years of trials, approval of eteplirsen.