The Countdown has begun…Approval for eteplirsen

Posted by: Christine McSherry on July 23, 2013




Last Friday afternoon, the three of us (Jenn McNary, Mindy Leffler and myself) had our seventh conversation with the FDA Director of CDER – Dr. Janet Woodcock. As always, Dr. Woodcock was warm, compassionate and extraordinarily supportive of our position – as parents of young men who have Duchenne.  She opened the conversation by acknowledging “time”, that 6 months have quickly passed since we started these conversations, she knows that “time” is not on our side.

The conversation covered a variety of topics – but we concentrated on discussing ways to “shave off time”.  How can the approval process go faster, is there flexibility in manufacturing, are there rumblings about dystrophin as a surrogate endpoint, can we make the next future trials in exon skipping – happen faster, do we need a panel for approval…please tell us Dr. Woodcock, how can we make this go faster – lives, thousands of lives can be changed…our boys included.

Dr. Woodcock carefully explained all the protocols and processes of getting drugs approved, always highlighting the expedited paths for conditions that have “unmet medical need”.  There was no point in the conversation, although she  had  plenty of opportunity, did she try to discourage or caution against our strong position for approval, rapid approval of this compound.

As we sit back now, and wait – we know that we have done everything possible that could have been done.  All three of us added vital and important data to the packet – the perspective of a parent, as well as the additional color to the already solid scientific packet from the sponsor.  We walked the FDA through “permissible risk”, met with Dr. Hamburg, we brought testimony from parents who have boys in the trial to dispel any thoughts that the boys were not stabilizing – or that the boys were outliers, we showed an hour long video presentation of “gains” – showing how the drug improved activities, like hiking, walking the dog, mowing the lawn – the things that the sponsor can not capture quite as well as a parent –  the everyday things that are important to the boys.  We educated the public and community about dystrophin, why it is important to this trial,  and the risk associated with not having it accepted as a surrogate, the risk of a delay in other exon skipping trials.  We read and studied the natural history papers published in Duchenne, exon skipping – and just about everything else when it comes to drugs and trials in Duchenne, we talked to experts, educated ourselves in FDA policy, met with former FDA agency members, spoke and worked with experienced health care lobbyists. We held a Congressional Briefing – standing room only – for members of Congress and their staffers to learn more about Duchenne and the recent developments, including our interactions with the FDA.  As a result, a Dear Colleague letter from both the House and the Senate was circulated, nearly 40 members total signed on and pledged to support the FDA in making a positive decision. We garnered over 180,000 signatures through change.org, kept the noise level up in the press (Today Show, WSJ, Politico and Roll Call) and leveraged the use of social media to get our message out. Other parents joined us along the way, and gave incredible support, connections and helped us spread the message.  We sought out opportunities at conferences and meetings where we knew our perspective would be heard – and we kept the pressure on.

As parents, and newly formed “advocates” – we also withstood a fair amount of criticism, but that is to be expected, and we would not back down.  Change does not come easy or without a price – but in our hearts we know this change is good and for all the right reasons.  Never did any one of us fight for the spotlight – each of us valued and respected one another, we left our egos at the door – and worked for our boys – all the boys.

The agency has all the information they need.  The data provided by the sponsor – stands on its own, the compound is safe and efficacious, any chatter that tried to once dispel that, is now gone. It is up to the Drs. Woodcock, Temple and the Division of Neurology now.

Last night I had a dream. Jett fell out of his power-chair, and instead of just lying there, he sat up and scooted on the floor. I chased him to help, and then found him trying to walk with crutches…it was only a dream, one that does not stand a chance of becoming a reality – but in my dreams, if only for a moment, it is a reality – the reality that we will win this fight, the fight that allows him to live longer.

News could come by the end of July – or later.  After listening to Dr. Woodcock on Friday, I am more optimistic it will come soon . However long, I will fight, we (Jenn, Mindy, myself and the entire Duchenne community) will keep fighting, because they are our sons’…and that is what we are suppose to do.




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