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With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

A top Food and Drug Administration official on Friday again advocated for the speedy approval of gene therapies for rare diseases. But he didn’t drop any clues on where the agency stands on a coming decision to possibly broaden use of one of them, a Duchenne muscular dystrophy treatment the regulator cleared last year.

At a meeting hosted by the patient advocacy group CureDuchenne, Peter Marks, head of the FDA office that reviews gene therapies, said the agency’s thinking has changed in recent years to become more patient focused. That mindset has led it to more aggressively look for ways to speed the development of rare disease gene therapies.

“Although we’re a regulatory agency,” he said, the regulations “have to ultimately serve getting products to patients. So we’re trying to focus on the patient, and use that to negotiate the regulations to get there as rapidly as possible.”

Those comments build on points Marks has made before. Last year, at a meeting held by a different advocacy group, he advocated for flexibility in reviewing rare disease gene therapies while fighting off criticism about accelerated approvals, which allow drugmakers to bring therapies to market based on interim measures of benefit. On Friday, he again threw support behind speedy clearances, noting that they are a “very important” tool in bringing forward rare disease treatments.

“There are some that don’t like this pathway, because for larger indications, they view it as a way of getting around the FDA’s need for substantial evidence of effectiveness,” he said. But in rare diseases, “we need something now, not later, so if we can bring things up and move them up with this pathway, it’s important to use.”

Marks spoke little about Sarepta Therapeutics’ Elevidys, the first approved gene therapy for Duchenne. Elevidys is currently only available for certain 4- to 5-year-old boys under an conditional approval granted by the FDA last June. By June 21, though, the agency could convert that clearance to full approval and authorize use more widely.

Sarepta’s case isn’t clear-cut. The company has asked the agency to approve Elevidys for all people with Duchenne and a confirmed mutation to a certain gene. It’s made the request even though the therapy last October failed the main goal of a trial meant to verify Elevidys’ benefit. The company is leaning on other supportive evidence, and analysts expect the FDA to endorse its case.

One reason analysts are optimistic is because the regulator didn’t reconvene an advisory panel that only narrowly recommended approving Elevidys last year. Another is the influence of Marks.

Last year, Marks reportedly pushed the FDA to schedule an advisory meeting after learning FDA scientists were inclined to reject the treatment, according to Stat. Afterwards, he overruled agency reviewers in granting an accelerated approval to the therapy in certain 4- to 5-year-old boys. Marks was also one of the FDA officials who spoke with Sarepta following the negative confirmatory trial results in a meeting that Sarepta executives later described as “very encouraging.”

Marks’ comments at Friday’s meeting are therefore notable. There has been “high focus on Dr. Marks’ tone, and whether there may be any hints about how the FDA is thinking,” wrote RBC Capital Markets analyst Brian Abrahams, in a Friday note to investors. Yet “somewhat surprisingly, [he] was a bit hard to read when it came to Elevidys.”

In his remarks, Marks pointed to a slide showing Elevidys’ results in 4- and 5-year-olds, noting those findings “allowed us to give this an accelerated approval” even though the overall study population “did not have the kind of statistical significance that we needed to see.”

“In the coming months, we’ll hear more about” the pending application, he said, adding that once the FDA sees “the clinical endpoints they need” it could convert Elevidys’ clearance to full approval.

Abrahams speculated Marks’ commentary suggests the “totality of trends” Sarepta showed in testing may be enough for the agency to consider at least an accelerated approval in older Duchenne patients who can still walk.

“We still do think Sarepta is likely to receive an approval,” Abrahams wrote. “That said, his specific language” was “a bit more opaque than we might have expected.”