How you can help…Give them more TIME

Posted by: Christine McSherry on November 17, 2013

Time – again, that word that makes me watch the clock all night…the word that makes me anxious for the early morning hours to come – so I can start my day.  My day that will be filled with looking for more time – time for me to find more time for Jett.  How ironic. That word has shaped the last decade of my life…the hours that turn into days, weeks and now years. And now the time is here. There is a safe, effective drug that could give Jett more time. But last week, the FDA concluded that they needed more information, and of course more time.

As I tried to summarize the past week’s events, between the conference calls, the emails, the direct messages – and trying to take care of my family – I am going to give you Tracy Seckler’s summary.  Tracy is co-founder of a great Duchenne organization, Charley’s Fund.  Charley is 13 – and while he will not benefit from eteplirsen, he will benefit from the other compounds that are in the exon skipping pipeline.  Tracy realizes that if we are not successful using this regulatory pathway for eteplirsen, then our chances for using it with other compounds in Charley’s lifetime or slim to none.

Thank you in advance for taking the time to read,  please check back – we will have additional requests from the community to help in our effort.


In Tracy’s words:

Yesterday Sarepta, the company developing this drug, announced that the FDA does not encourage submission of a New Drug Application (NDA) at this time. This is a direct turnaround from the FDA’s position just three months ago, when, according to Sarepta, they told the company they would accept an NDA. According to the press release Sarepta distributed yesterday morning, the FDA raised many questions about the clinical trial data and about this therapeutic approach called exon skipping.

The FDA’s change of position was most likely influenced by the recent failure of a different drug produced by Glaxo Smith Kline called Drisapersen. Like Eterplisen, Drisapersen is also designed to produce dystrophin via exon skipping, but a large clinical trial of Drispaersen did not result in stabilization or improvement in hundreds of boys. The FDA said those results call into question whether dystrophin production will lead to clinical benefit. But here’s the thing: No one knows if the kids in the failed Drisapersen trial were producing dystrophin, and if they were how much of it was found in the muscle fibers. We don’t know because GSK hasn’t released that data.

If we are to compare competitive products, the recent failure of Drispaersen should buttress Sarepta’s case, not hurt it. Drispaersen was close to what our children need, but not quite right. Due to toxicity issues, the drug could not be dosed at high enough levels to have across-the-board efficacy. In contrast, Sarepta’s drug has a stellar safety profile. In two years, not one drug-related adverse event has been reported.

This is a lot to take in, I know. But it can all be boiled down and stripped away to these essential points:

1. Eteplirsen is safe. It is possible that unknown side effects may appear once children are on the medicine for longer than two years. But every single family I know is more than willing to take that risk. Most of us currently treat our boys with poisonous steroids that result in awful side effects, just to give our children two years’ additional walking and breathing time.

2. Eteplirsen may not work. An N of 12 is certainly not a large-scale, fool-proof data set. Once hundreds of children are taking the drug, we may learn that the dystrophin production and functional benefit we saw in the 12 boys was a fluke. Again, every family I know is more than willing to take that risk. I am putting my money down that Max can now march in the Halloween parade and Billy can now push his mom’s shopping cart when they could not do those things before starting Eterplisen treatments because they are now producing dystrophin, not because all of a sudden they just happen to be feeling great.

3. We need to find out if this drug is the real deal. The FDA can move forward from here in two ways: approve it now on an accelerated basis because it produces dystrophin, and LACK OF DYSTROPHIN CAUSES DUCHENNE. Or, require longer, larger studies that will cost millions of dollars and – more important – many children’s lives. As of yesterday, it looks like the FDA is choosing the latter. As a community, people impacted by Duchenne must come together to reinforce the reasons the FDA must consider moving forward with an accelerated approval. Our children’s lives depend on it.

Take it from my friend Carl, who is 26 and has Duchenne. If anyone knows the stakes, he does:

You do not need to get into any technical details. The bottom line is this: the safety data to date is perfect and the efficacy data is promising. A drug with good safety and efficacy data for a disease with NO treatment approved like Duchenne should not be DELAYED due to failure of other drugs in trials!

PLEASE HELP – copy and paste this letter into an email and send to the Regulatory Officials listed. 
Dear Friends,

We write to express our frustration and sadness. We write to express our solidarity with the entire Duchenne community. We write to ask you to understand that now, more than ever, our community must stand together, we must speak truth to power and we must believe that we can affect change. The FDA made a mistake earlier this week with its seeming reversal on Serepta’s NDA application.

We know that is true because Eterplisen has a great safety profile.

We know that is true because Duchennne has taught us what permissible risk means.

We know that is true because we understand that a three year delay in getting a drug to a patient with Duchenne could kill that patient.

We know it’s true because Eterplisen helped 12 boys produce dystrophin.

We know it’s true because, when we look into our own kids eyes every day, we see their dreams and ambitions reflected back at us. Duchenne destroys muscles, but our children will dream until the day they die.

We know it’s true because, at the end of 96 weeks, all ambulatory children in the study had either improved or stabilized in 6 minute walk-test.

Mostly we know it’s true because there is nothing more unbearable as a parent than seeing your child’s eyes looking to the future that you know may not exist.

Eterplisen offers many kids with DMD the possibility for a future. Every indication, up until a few days ago was that the FDA agreed with this assessment. Every indication was that the FDA understood the extreme nature of Duchenne. We believed as a community that the FDA would approach other potential Duchenne drugs with the same common sense and compassion. Our fragile hope, based out of love for our children, had Serepta’s and positive and indisputable data to back it up.

The Duchenne Alliance was founded on hope but also on the core belief that together, we are stronger. We are a coalition of over thirty Duchenne focused organizations from across the globe who will continue to seek out, to fund, lobby and advocate for a treatment for every child with Duchenne. In the meantime, it is time for us to put our belief in collaboration to work. We are calling on the entire community, patients, parents, friends and representatives in government to demand that the FDA reconsider its decision regarding Serepta’s NDA application.

What can you do?

• Please send the following letter to your representatives in congress and to the email addresses below.
• In order to demonstrate your solidarity with the families and patients who have been impacted by this decision, change your FB profile pic to the Duchenne Alliance hourglass.

Dear Drs. Woodcock, Jenkins, Temple, Unger, Bastings and Farkas,

I understand that the FDA has reversed its previous stance on whether to consider Sarepta’s NDA for Eterplisen. The Agency has cited the recent failure of other dystrophin-producing drugs as one reason to require that Sarepta conduct a large, placebo-controlled phase 3 study before applying for approval. I do not understand this reasoning, as we don’t even know how much, if any, dystrophin was produced by the drugs that failed to meet their primary clinical trial endpoints. In contrast, we know that Eterplisen produced dystrophin in all 12 treated patients at levels widely believed to lead to functional benefit. What’s more, the treated patients have stabilized since the dystrophin took effect. One third of the children actually improved, a clinical outcome that is unheard of in Duchenne.

I do not have a rare fatal disease, but if I had one I would want the freedom to choose a drug in consultation with my physician that has a highly favorable safety profile and early indications of efficacy. I believe the FDA’s #1 job is to protect people. While we understand there are certain risks with approving a medication studied in only 12 patients, the risk is a minimal and tolerable one compared to the certain pediatric deaths and losses of ambulation that will occur if you require several more years of study before approving this drug.

I implore you, work with Sarepta to accept the creative and aggressive confirmatory trial design they have planned. Understand that children will die and our kids will lose their ability to walk if this decision continues to be postponed.

Insert your name here

Send the e-mail to the following FDA officials:

Be sure to cc these people:

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