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 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.
Our Letter to the FDA:
October 27, 2014
William Dunn, M.D
Director, Division of Neurology Products
Center for Drug Evaluation and Research
Food and Drug Administration
10903 New Hampshire Avenue, Bldg 22, Rm 4338 Silver Spring, MD 20993
Dear Dr. Dunn,
This morning we were invited to participate in Sarepta’s call regarding their press release surrounding a regulatory update on eteplirsen. We learned that Sarepta will delay submission of their NDA until mid-year 2015. As you might imagine, our Duchenne community is devastated by this news.
During the call we understood from the company that the agency is now requesting more safety data, patient-level natural history data, and an independent review of the biopsy data used to calculate dystrophin levels.
We further understand that many within the community have agreed to comply with the Agency’s request to undergo a fourth muscle biopsy. While we understand the potential for value to both the approval process and subsequent reimbursement authorities, the fulfillment of this request requires great personal sacrifice on the part of trial participants – children. This is further demonstration that our Duchenne community is willing to do whatever it takes to expedite safe and effective therapies; we urge the Agency to demonstrate the same level of flexibility in review.
We are terribly disappointed by this news, but are hopeful that the additional information you have requested will allow you to gain sufficient insight into this rigorous review of safety and efficacy, leading to an approval of eteplirsen. We further hope that the rigor of your review and the comprehensiveness of the data package being provided by the sponsor will lead to willingness at the Agency to consider accelerating follow-on exons in a more expeditious manner. As a community we believe eteplirsen to be a foundational therapy, restoring dystrophin and significantly slowing disease progression. I know the agency is well aware of this critical unmet need and with us, shares an urgency to deliver approvals so that these young men can live productive and long lives.
We appreciate your need for rigor, but want you to know that the Duchenne community believes the benefit and risk equation is well documented. Parents’ first priority is stabilizing disease progression. We look forward to the mid-2015 submission and approval of eteplirsen and the consideration of approval of PMO as a class of drugs that will have the ability to slow/halt progression in a great many of the individuals living with Duchenne.
President and CEO
Parent Project Muscular Dystrophy
cc: Ellis Unger, MD; Robert Temple, MD; Ronald Farkas, MD