Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA…
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Today’s press release and investor call from Sarepta (http://bit.ly/1xwCbi1) have sent a ripple of angst and sadness through our community.
Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.
As a community we believe exon-skipping drugs like eteplirsen hold potentially life-altering benefits, and must be moved forward as rapidly as possible. In addition, we believe eteplirsen to be a critical foundational therapy, restoring dystrophin and significantly slowing disease progression. We look forward to the mid-2015 submission and approval of eteplirsen and the consideration of approval of PMO (phosphodiamidate morpholino oligomer) as a class of drugs that will have the ability to slow/halt progression in a great many of the individuals living with Duchenne.
PPMD has also reached out to leadership at Sarepta to support the pathway forward for these trials and to work to ensure the approval and availability of an effective therapy to our Duchenne community at the earliest moment possible. As always we will report back to you with updates on next steps and anything we as a community can do to help this process.