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This weekly column offers opinions on the latest pharmaceutical industry news.

An advisory panel to the Food and Drug Administration was supposed to have met last Friday to decide the fate of an experimental treatment from Sarepta Therapeutics for a rare disease called Duchenne muscular dystrophy. But with a blizzard engulfing the nation’s capital, the agency delayed the meeting — and, in so doing, left alive the last glimmer of hope for families afflicted by this fatal genetic disorder.

They may have a long wait. When the FDA panel finally meets, it appears likely it will recommend against approving the drug from the Cambridge, Mass.-based company. And under the circumstances, this might be a good thing.

Of course, no one wants to see children suffer, and it would be a huge boon to patients and society for a therapy to become available for a critical unmet need.


But there is considerable doubt about whether there is sufficient evidence to warrant approval. And bowing to parental pressure would undermine the integrity of the FDA and its mandate to keep patients safe.

“Patient input is important,” said Daniel Carpenter, a political scientist at Harvard University who studies the FDA. But an approval in this case “risks converting the FDA review process into a [political] party primary, where only the most extreme voices show up.”


It’s been a particularly tough month for the families of children with Duchenne. First, the FDA rejected a drug from BioMarin Pharmaceutical that works in much the same way as the Sarepta treatment. Both are designed to splice out a section of faulty genetic code to create a working version of a missing muscle protein in patients. In reaching its decision, the FDA determined on Jan. 14 that BioMarin’s trial data failed to prove a clinical benefit. A day later, the agency published an internal review of the Sarepta drug, eteplirsen, expressing “significant concerns” over trial design and saying that some measures of efficacy “appear unreliable.”

Patient advocates are hoping that when the FDA panel of outside experts eventually meets — a rescheduled date has not yet been set — the committee will take a more favorable view of the Sarepta drug than that of the agency staffers.

“The data does suggest there is a response to this drug, and the FDA knows we need options,” said Pat Furlong, who heads Parent Project Muscular Dystrophy, the largest DMD-focused nonprofit organization in the United States.

“There is a lot at stake,” agreed Manu Gambhir, an Internet entrepreneur in Philadelphia, whose 14-year-old son is enrolled in a Sarepta clinical trial. “The concern is another rejection could have a chilling effect on further investment in this field,” he said. “If that happens, lives may be lost.”

Listen to the Signal podcast: The corporate saga of the Duchenne drugs

Sarepta took the unusual step of filing a rebuttal to some of the concerns raised by the FDA reviewers. Nonetheless, the drug maker must still contend with the fact that its data were generated from a very small study of just 12 boys. (The disease almost always affects boys.) Such studies can be a red flag, because trials of this size may fail to reveal all the ways in which a drug may later cause unforeseen reactions in a larger population of boys who are prescribed a treatment.

“This small data set is part of the problem,” said Simos Simeonidis, an analyst at RBC Capital Markets.

“But even if the FDA rejects the drug,” Simeonidis added, “I don’t think investment in that area is going to go away.”

After all, some three dozen companies are currently developing some type of treatment for Duchenne. And the lure of meeting an unmet medical need is unlikely to waver, given the high prices that can be charged for such specialty medicines.

To be sure, FDA officials have been sympathetic to Duchenne patients, meeting numerous times with parents of boys with the disease, and the agency even used a regulatory guidance drafted by Furlong’s advocacy group as a blueprint for recommendations on developing drugs for the rare disease.

Yet there is a decided difference between being more responsive to patients and lowering the threshold for drug approvals to mollify distraught families.

If there is uncertainty about the benefit of the Sarepta drug, FDA officials should demur, wait to see more trial data, and reject the medicine for now.