Organization Urges FDA to Use the Evidence Presented by Dozens of Scientists, Physicians, Patients and Advocates as Required by 2012 FDASIA Act for Conditional Approval

Experts Remind Duchenne Community that Ad Comm Vote on accelerated approval was very close (6 yes-7 no); FDA Can Still Make Right Decision to Approve the Drug

Physicians and scientists who have studied Duchenne muscular dystrophy and treated patients for decades presented compelling evidence that a new Duchenne drug, eteplirsen, works during the open public hearing section of a U.S. Food and Drug Administration (FDA) Advisory Committee Meeting (Ad Comm). They were part of an unprecedented crowd of more than 800 people imploring the FDA panel to see past rigid rules created for mass population drugs and listen to the science supporting this rare disease drug’s safety and efficacy as required by the Food Drug and Safety Innovation Act passed in 2012.

“We thank the scientific community from around the globe who came to Washington DC to stand with our community and defend the science behind eteplirsen. However, we are deeply concerned that the votes at the Ad Comm displayed a lack of understanding of the science and the impact that this investigational treatment has and could have on the children who are living with this fatal disease. We encourage the FDA to honor FDASIA, and fully utilize the tools Congress has given them,” said Race to Yes co-founder Marissa Penrod of Team Joseph.

“Monday’s advisory committee meeting on eteplirsen saw a unified show of support from the patients and scientists who live with, study and treat Duchenne. The testimony from Duchenne experts during the open public hearing clearly underscores the positive data generated in the small clinical trial. Scientists from Harvard, Stanford, Northwestern, Johns Hopkins and other esteemed institutions traveled from around the world to present the facts. Every single one of these scientists supports this drug’s approval,” said Race to Yes co-founder Tracy Seckler of Charley’s Fund. “On Monday, Dr. Janet Woodcock, the senior FDA official whose division is ultimately responsible for the decision, highlighted the importance of applying flexibility and considering public input in cases of fatal diseases with no treatments. She also acknowledged the grave consequence of failing to approve a drug that works. We are confident the FDA will make the right decision.”

Under FDASIA, Congress gave the FDA the authority to grant accelerated approval for new treatments for life-threatening rare diseases precisely in cases like eteplirsen. Under FDASIA rules, FDA has a duty to hear and consider the evidence and knowledge available from specialized experts and the community, as well as the drug maker. It also has the flexibility to consider scientific clinical trial methods appropriate for small patient populations racing against the clock like children with Duchenne.

The Race to Yes noted that the Ad Comm panel’s key vote on accelerated approval was split 6-7. Panel members were clearly constrained by the wording of the voting questions. One AdComm panelist, in reference to one of the other voting questions, went so far as to state at the meeting, “Based on all I heard, the drug definitely works, but the question was framed differently.”