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As a crucial deadline nears for a closely watched regulatory decision, two Republican senators are urging the Food and Drug Administration to approve a controversial drug to treat Duchenne muscular dystrophy.

In a letter sent last Friday, the senators expressed “disappointment” that an FDA advisory panel last month voted not to recommend eteplirsen to combat the disease, which is a rare and fatal genetic disorder that causes muscles to waste away. About 13,000 children, mostly boys, are afflicted and they typically die before turning 25 years old. The panel determined the drug was not effective.

The FDA is scheduled to decide whether to approve the drug, which is made by Sarepta Therapeutics, by this coming Thursday. And its decision is being closely watched as a litmus test for the agency, which is grappling with increasingly assertive patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs.


In this instance, patient advocates and their many backers want the FDA to use what is called the accelerated approval process to endorse eteplirsen. This approach relies on a substitute outcome in a clinical trial to suggest a drug may have, but does not guarantee, a benefit.  The senators maintain that accelerated approval was designed for circumstances such as those posed by eteplirsen.

“For a disease like Duchenne muscular dystrophy and other diseases that are highly debilitating and almost certainly fatal, we hope you will employ these flexibilities and considerations for the maximum benefit of patients who have no other alternative,” wrote Ron Johnson, a Wisconsin Republican who chairs the Senate Committee on Homeland Security and Governmental Affairs, and Dan Coats, a Republican from Indiana, in their letter to FDA commissioner Dr. Robert Califf.


The missive is only the latest such attempt to sway the agency. The same lawmakers previously wrote the FDA to urge approval. And earlier this year, more than three dozen medical experts, some with ties to Sarepta, sent a letter in which they chastised FDA staff for pooh-poohing clinical evidence that the drug works. They argued that the totality of clinical trial data provides “substantial evidence” that the medicine is effective.

But FDA medical staff has taken a dim view of the drug. At issue is whether eteplirsen can sufficiently produce higher levels of a protein called dystrophin. Without this protein, muscle fibers degenerate and voluntary movement becomes impossible. But FDA staffers questioned the validity of a 12-patient study, which was not run according to the usual standards. They also expressed doubts about whether the drug produced dystrophin and the viability of six-minute walking tests that trial participants underwent.

Nonetheless, the agency has also sent mixed signals. Janet Woodcock, who heads the FDA Center for Drug Evaluation and Research, took the unusual step of not only attending the advisory panel meeting last month, but also offering remarks. To some, she seemed to suggest that agency officials are considering more than just the scientific data that was criticized by the FDA’s own neurology division.

The agency, she said, has “flexibility and that’s where we should take the views of the (patient) community into account … It’s possible to reach different conclusions based on the data presented today … Failing to approve a drug that actually works in devastating diseases — these consequences are extreme.”

Her remarks were made before an overflow crowd that included hundreds of parents and their children, some of whom appeared in wheelchairs. In fact, only one of the dozens of people who spoke during the public session, argued against approval.

The advisory panel, which consisted of scientists and doctors who are not FDA employees, voted seven to three, with three abstentions, against recommending approval. But asked whether accelerated approval should be granted, the vote was much closer vote: seven said no and six said yes. The agency is not obligated to accept panel recommendations, but usually does so.

In their latest letter, the senators argued that the way the FDA framed the questions may have made it more difficult for some of the panel members to vote in favor of the drug. They then go on to note that the issues transcend this one drug, but can be applied to other situations where patients are clamoring for life-saving medicines and need a more flexible FDA.

“Patients are crying out for the FDA to hear them,” they wrote. “They are engaged and knowledgeable and only want the agency to do what is already within their power.”

Meanwhile, some Wall Street analysts believe the agency is unlikely to approve the Sarepta drug, although some also acknowledge that accelerated approval remains a possibility. The “FDA has gone to unprecedented lengths to give transparency into its evaluation process and actually seems to want to help the community as much as they are allowed to by their mandate,” wrote Cowen analyst Ritu Baral in a recent investor note.

The story has been corrected to state that Senator Dan Coats is from Indiana, not Iowa. 

  • The FDA is killing people. There are many drugs in the pipeline for terminal diseases that the FDA lets languish in the archaic and outdated three phase clinical trial design. It takes 10-15 years to get a drug on the market. Time that millions of people just don’t have. If a drug passes a phase 1 safety trial then terminal patients with no other treatment options should be allowed to try it. People are getting fed up. There is tremendous pressure from patient groups that are demanding the FDA change and become more reasonable. It makes no sense that approval for a drug that treats a terminal disease and an acne drug should travel through the exact same approval process. This is why Right To Try is now approved in 28 states and more on the way. And there is a national RRT bill making its way through congress. The FDA is on notice. You will change willingly or you will change by force. But you will change.

    • DL, a little screed is good for the soul – hopefully you have been helped. And now, with apologies to our scribe and out hosts, I continue:
      DL, ii puzzles me why you end your … words with this ‘You will change willingly or you will change by force. But you will change.’ The FDA website is in a different domain – but I suspect you choose to splash your message here rather than there for some good reason.

      The subtle dog whistle you are using is slander IMHO. ‘The FDA is killing people….millions of people’ Call me a denier but where do you get your numbers? The great news for you is that freedom of speech prevents the government, the FDA, from taking umbrage. You have the freedom to bloviate, others have the freedom of speech to quote the Bard – a tale ‘Told by an idiot, full of sound and fury, Signifying nothing.’

  • The only guaranteed benefit from this medication is the coffers of Sarepta swelling by pushing this for a subset at orphan-drug price$ – 200K and up. Which will open a flood of ‘off-label’ scrips because ‘we don’t know that it won’t work.’ As I have said before, let them pledge to sell it at even $3K/month (so only $60 million) and I will apologize for misjudging them.

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