FDA delays decision on eteplirsen

Sarepta Therapeutics announced this morning that the FDA has indicated it will not reach a final decision on eteplirsen by the May 26th deadline.  We understand and share the frustration many of you feel that the FDA still hasn’t made a final decision on eteplirsen despite strong scientific evidence that supports approval. We have already waited far too long.

For the past several years, the Duchenne community has helped lay the groundwork for the accelerated approval of eteplirsen.  We have encouraged the FDA to follow FDASIA and to make good on its commitment to take patient input into account in the drug development and review process.  More people attended the eteplirsen Advisory Committee meeting than any other AdComm in history.  And eteplirsen has garnered unprecedented scientific and medical support.

While we acknowledge that a delayed potential approval is better than a rejection tomorrow, one thing is certain: no boy or girl with Duchenne should have to wait through years of delays for access to a safe, effective therapy.

We will continue to focus on the strength of eteplirsen’s safety and efficacy data, on the broad and unprecedented support from Duchenne researchers and clinicians, and on the flexibility and tools that the FDA has been entrusted with to approve safe therapies that treat severely debilitating and life-threatening illnesses with an unmet medical need.

We remain hopeful that the FDA will do the right thing and approve eteplirsen, and that they do so quickly – because those affected by Duchenne are declining every single day. We will continue to proclaim this simple fact:  the science supports approval of eteplirsen.

Thank you for your continued commitment.  We will keep you updated as we learn more.