WASHINGTON, April 9, 2014 — Parents of children facing death from Duchenne muscular dystrophy today asked the U.S. Food and Drug Administration to approve in 30 days a well-tested drug that halts the disease with no side effects.
The parents, representing The Race to Yes campaign, challenged FDA Commissioner Margaret Hamburg to take the steps needed to approve the drug – eteplirsen – following positive comments she made to the news media on Friday, April 3 while appearing at a biotech conference in Boston.
“We are now at the point where this generation of children could be the last to die from Duchenne or the first to survive. We fervently hope it’s the first to survive.” parents Tracy Seckler and Marissa Penrod said in a letter to Hamburg. “Your comments make it clear that approving safe and proven Duchenne therapies such as eteplirsen is a priority at the FDA.” Both women are parents of a son with Duchenne.
Duchenne muscular dystrophy is the world’s leading genetic killer of children. An estimated 1 out of 3,500 boys has the deadly disease. As their muscles fail due to the lack of a key protein, its victims usually die in their teens and early 20s. Until now, no drug has been proven to effectively halt the progress of the disease. Long-term testing has shown eteplirsen enables the body to produce the protein – dystrophin –and stop the progress of the disease with no side effects.
The Race to Yes campaign asked Hamburg to move quickly to make eteplirsen available to boys whose genetic makeup shows they are amenable to it. After 17 months of delays by the FDA the national campaign organized to gather 105,795 signers on a White House petition in the 30 days between Feb. 27 and March 29. Signers urged the FDA to use its accelerated approval process to finally approve eteplirsen. The White House has yet to respond to the petition.
“This has been a long and laborious process,” they wrote, describing the time it has taken to put the drug’s producer, Sarepta, in position to make the drug available. The FDA, the parents contend, has been slow to provide the company the guidance needed to move the approval process forward. “The actions we request will resolve those issues.”
Those actions include encouraging Sarepta to submit a “new drug application” so “any outstanding questions regarding efficacy can be reviewed by an expert panel.” While approval is being considered, a new clinical trial should begin immediately so children who can benefit from eteplirsen can get on the drug without further delay.
Finally, the parents asked the FDA to “communicate a clear path toward approval” for other drugs based on the “same backbone chemistry as eteplirsen.”
“Eighty percent of children with Duchenne are waiting for follow-on compounds that are sitting on the shelf pending regulatory guidance,” the parents wrote.
Hamburg’s comments came in response to media reports in Boston that focused on two Duchenne families that are part of the campaign. Jenn McNary’s two sons, Max and Austin, have Duchenne. Max is doing well while receiving eteplirsen. Austin is amenable to the drug, but was not included in the long-term study. He is getting sicker. Parent Christine McSherry’s son Jett has been wheelchair-bound since age 13, and has since lost the ability to feed himself.
Text of the letter follows:
REQUEST FOR ACTION
Dear Commissioner Hamburg,
We want to thank you for your comments in a television interview in Boston last week making it clear the FDA places a high priority on approving safe and effective therapies for all children with Duchenne muscular dystrophy. This is an exciting moment in the history of this horrendous fatal disease. We are now at the point where this generation of children could be the last to die from Duchenne or the first to survive. We fervently hope it’s the first to survive.
Your comments make it clear that approving safe and proven Duchenne therapies such as eteplirsen is a priority at the FDA. We are confident the agency will move forward with the following three actions to ensure that children with Duchenne get the medicines they so desperately need without further delay. We ask that you take action on these items within 30 days:
1. Encourage Sarepta to submit a New Drug Application for eteplirsen so any outstanding questions regarding efficacy can be reviewed by an expert panel.
2. To allow children to get access to eteplirsen as soon as possible, agree on trial design for an open-label confirmatory study that can begin immediately.
3. Communicate a clear path toward approval for follow-on exon skipping drugs that are based on the same backbone chemistry as eteplirsen. Eighty percent of children with Duchenne are waiting for follow-on compounds that are sitting on the shelf pending regulatory guidance.
This has been a long and laborious process. Sarepta first requested a meeting with the FDA to discuss the implications of their positive Phase 2b data in December 2012. Nearly a year and a half later, the FDA still has not provided guidance on two key issues: use of the accelerated approval pathway and the design of a confirmatory study. The actions we request will resolve those issues.
Now that this matter has been brought to your attention, we are confident that with your leadership and your commitment to acting on safe and effective Duchenne drugs, the agency will act swiftly and decisively. We look forward to sharing your response with the 105,799 persons who signed our White House petition, members of the U.S. Congress, and the Obama administration.
On Behalf of The Race to Yes