Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a planned first-ever filing of an NDA for Duchenne.
PPMD applauds Sarepta Therapeutics for moving forward to file its application for eteplirsen, a potential therapy intended to skip Exon-51, and to seek Accelerated Review for this candidate therapy from the Food and Drug Administration (FDA) based on the results of its Phase IIb clinical trial. PPMD advocated for stronger Accelerated Review tools that more fully account for the needs of the rare disease community as part of last year’s debate on the FDA Safety & Innovation Act, and we urge FDA to use every tool at its disposal to conduct a swift and thorough evaluation.
Most recently, PPMD had a meeting with Dr. Janet Woodcock, head of the Center for Drug Evaluation and Research (CDER), during which we shared with her the results of our landmark benefit/risk survey of Duchenne parents to ensure the views of our patients and families are accounted for during the review of eteplirsen and other potential Duchenne therapies.
PPMD looks forward to continuing to work with leaders in government, industry and elsewhere to advance valuable Duchenne research, lengthen and improve the quality of life for every patient and to accelerate FDA review of potential therapies to deliver safe, effective and accessible to patients as quickly as possible.
This is a great day for the Duchenne community. Where at one time we saw nothing but a hopeless end, now we see nothing but endless hope. We are thankful to this community for all the work it has done together to raise awareness about the urgency to find treatments for all patients.