Posted by: Christine McSherry on April 16, 2013
Time, I talked to them about "time". How little of it I have for my son Jett. Jett is approaching his 18th year of life, he has Duchenne. His mutation is "out of frame, 45-50”. Jett would be eligible for eteplirsen.
In all the meetings, the multiple meetings, that I have had with regulatory officials, from the Shadow Health Minister, the EMA and MHRA in Parliament to the regulators at the FDA; Drs. Temple, Woodcock, Katz and the Commissioner, Dr. Hamburg - my constant message to them was to please help the sponsor, in the case Sarepta Therapeutics, to remove all barriers and obstacles that would take up additional "time" getting eteplirsen out to boys, like Jett, who need it.
What I heard in yesterday's press release and the additional information offered by Sarepta's CEO during a conference call, the regulatory agency is reacting and providing guidance in a very methodical and timely manner. They are requesting additional supportive information and are asking very important questions. They want to know more. They need additional clarity on something that has not been significantly measured as an endpoint. They are evaluating dystrophin as a surrogate endpoint - the underlying cause of the disorder.
They are anticipating using the safety data that was collected, up to 74 weeks, and data obtained in the next coming months to bolster any safety argument. Most importantly, the agency did NOT request a phase lll randomized controlled trial - instead, just the opposite.
Could this additional data requested, and the existing data set collected reduce the time to get eteplirsen approved, undeniably - yes. The information requested and provided by Sarepta, could be used toward the NDA – and reduce time. The agency is thinking ahead, they are being proactive in their approach, progressive and creative about moving this potentially life saving drug out in the most timely manner possible.
In the meantime, Sarepta has assured us that they will provide the FDA with all the supporting documentation, they will continue on their path to upscale manufacturing and the conversations will be ongoing with the agency to ensure they are all working together and in the right direction, in an effort to save time.
It sounds to me as if all the details of accelerated approval are being worked outahead of time. However, we still need to be aware, that little time slips add up and that is something the Duchenne community cannot afford. As a community, we need Sarepta and the FDA to be aware that there is limited time, both of them need to do everything possible to minimize anything that could increase time. On the call last night, Sarepta’s CEO clearly stated that the FDA was willing to be flexible in their schedule to expedite the process. We also heard this message of urgency in Sarepta’s CEO’s speech last week during the WODC conference.
As an advocate, a mother, and a member of the Duchenne community, who feels time weighing heavily on my shoulders right now, I can't let my foot off the gas, I will continue to advocate strongly for streamlining the process with continued communication between the agency and the sponsor. Now more than ever, time is truly of the essence, for Jett and many others who could benefit from accelerated approval.
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