FDA's Marks Advocates for Rapid Gene Therapy Approvals Ahead of Duchenne Decision

On Friday, a senior official from the Food and Drug Administration (FDA) reiterated the agency’s commitment to expediting the approval process for gene therapies aimed at treating rare diseases. However, he provided no clear indications about the FDA's stance on expanding the use of Elevidys, a Duchenne muscular dystrophy (DMD) treatment approved last year.

Peter Marks, who heads the FDA office responsible for gene therapy evaluations, spoke at a meeting hosted by CureDuchenne, a patient advocacy group. Marks emphasized that the FDA's approach has evolved to prioritize patient needs, resulting in a push for quicker development and approval of gene therapies for rare diseases.

"Although we're a regulatory agency," Marks stated, "the regulations must ultimately serve getting products to patients. So, we're trying to focus on the patient and use that to navigate the regulations to get there as quickly as possible."

Marks' comments echoed previous statements he has made advocating for flexibility in reviewing gene therapies for rare diseases. He has consistently supported the use of accelerated approval pathways, which allow therapies to reach the market based on preliminary evidence of benefit. On Friday, he reiterated the importance of these expedited approvals, particularly for rare disease treatments.

"Some people don't like this pathway because, for larger indications, they see it as circumventing the need for substantial evidence of effectiveness," he noted. "But in rare diseases, we need treatments now, not later, and if this pathway can expedite that, it’s crucial to use it."

Marks made limited remarks about Sarepta Therapeutics' Elevidys, the first gene therapy approved for Duchenne muscular dystrophy. Currently, Elevidys is conditionally approved for boys aged 4 to 5. By June 21, the FDA might grant full approval and expand its use.

Sarepta has requested approval for Elevidys for all DMD patients with a specific genetic mutation, despite the therapy not meeting the primary endpoint in a recent trial. The company relies on other supportive data, and analysts believe the FDA might endorse it.

Analysts are optimistic for several reasons, including the FDA’s decision not to reassemble an advisory panel that narrowly recommended Elevidys' approval last year. Additionally, Marks' past influence is seen as a positive sign. According to reports, Marks previously advocated for an advisory meeting when FDA scientists were inclined to reject Elevidys. He subsequently overruled agency reviewers to grant accelerated approval for the therapy.

Marks' comments at Friday’s meeting have thus garnered significant attention. RBC Capital Markets analyst Brian Abrahams noted that Marks’ tone was being closely scrutinized for any hints about the FDA’s thinking. However, Marks' stance on Elevidys remained ambiguous.

During his presentation, Marks referenced Elevidys' results in 4- and 5-year-olds, stating that these findings justified the accelerated approval despite the overall study not achieving the necessary statistical significance.

"In the coming months, we'll hear more about the pending application," Marks said, indicating that full approval could follow if the FDA finds the clinical endpoints satisfactory.

Abrahams speculated that the trends observed in Sarepta's testing might be sufficient for at least an accelerated approval in older patients who can still walk.

"We still believe Sarepta is likely to receive approval," Abrahams wrote, "but his specific language was more opaque than expected."

In summary, the FDA remains dedicated to fast-tracking gene therapies for rare diseases, emphasizing patient-focused regulation. The future of Elevidys' broader approval remains uncertain, but signs suggest optimism among analysts.

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