T

he saga surrounding the fate of a controversial Sarepta Therapeutics drug grew even more intriguing today after the US Food and Drug Administration confirmed that a key staffer who opposed approving the medicine has left the agency.

The news sent shares in Sarepta soaring as investors began betting the departure of Dr. Ronald Farkas, who led the clinical team in the neurology products division, might signal the agency was now leaning toward approving the medicine, which has been developed to treat Duchenne muscular dystrophy. At one point this morning, Sarepta stock was up as much as 26 percent.

An FDA spokeswoman would not say when Farkas left, and whether his departure is tied to the review for the Sarepta drug is not known. Farkas could not be reached for comment. But he has played a key role in the review process. At an FDA advisory meeting last April, Farkas told an expert panel the agency had “strong doubts” about the viability of a clinical trial the company is relying on to win regulatory approval (here is the agency review). The panel voted against approval by a slight 7-to-6 margin.

advertisement

His departure comes amid increasing speculation and strong emotions about an FDA decision. The disease destroys muscle fibers and eventually confines boys to wheelchairs before sending them to an early death. There are no treatments currently available, and the FDA has rejected two medicines from two other companies, prompting parents to worry that their children will be left without options.

As a result, the drug has become an important litmus test for the agency as it balances intensifying demands to approve more drugs more quickly — especially for rare diseases — and the need to maintain scientific standards for endorsing medicines. Some describe the battle as the biggest clash between regulators and patients since the AIDS crisis more than three decades ago.

The FDA, however, has been accused of unnecessarily blocking approval, given the unmet medical need. Over the past year, a growing number of Washington lawmakers — spurred by anxious parents — urged the agency to greenlight the drug. And more than three dozen medical experts, some of whom have ties to Sarepta, maintained that FDA reviewers made several blunders in their initial review of the drug.

In a 115-page report released earlier this year, though, the FDA medical review team led by Farkas raised doubts about the veracity of a 12-patient clinical trial that Sarepta relied on to make its case. They also questioned the viability of six-minute walking tests that trial participants underwent. The data, they wrote, “did not provide statistical evidence” to say that the drug is effective.

His opposition, in fact, has been seen as a key impediment to agency approval. Earlier this year, editorials that appeared in the Wall Street Journal singled out Farkas as the main obstacle. Citing various steps in the review process, the editorials concluded that Farkas and another FDA reviewer had “lost the public’s confidence that they can conduct a fair review.”

Meanwhile, at the meeting last April where Farkas expressed concerns about the Sarepta trial data, a high-ranking FDA official — Dr. Janet Woodcock, who oversees the agency division that is responsible for approving drug medicines — attempted to appease the large crowd of DMD parents and their supporters. “It’s possible to reach different conclusions based on the data presented today,” she said.

Sign up for our Pharmalot newsletter

Please enter a valid email address.

To some, the FDA appears to be sending mixed signals. It hasn’t helped that the approval process has dragged on, further fueling speculation about the outcome. Last winter, the agency postponed an initial advisory committee meeting for the drug, which later took place last April.

But in June, the agency asked Sarepta to submit more data about its clinical trial, which some have interpreted as a sign that FDA officials are searching for ways to approve the drug. Such  a move would underscore the political sensitivities surrounding this particular medicine and the potential for a clash inside the agency over the approval standards.

— This story has been corrected. Dr. Ronald Farkas was previously identified incorrectly as Dr. Robert Farkas. 

Leave a Comment

Please enter your name.
Please enter a comment.

Recommended Stories

Sign up for our biotech newsletter, The Readout

A guide to what’s new in biotech — delivered straight to your inbox every weekday morning.