Race to Yes Urges FDA to Approve Eteplirsen to Treat Duchenne Muscular Dystrophy

Özge Özkaya, PhD avatar

by Özge Özkaya, PhD |

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FDA and Duchenne MD drug

The Race to Yes initiative is urging the U.S. Food and Drug Administration (FDA) to carefully consider the evidence presented at the latest Advisory Committee meeting and to approve fast access for the Duchenne muscular dystrophy drug, eteplirsen.

“Monday’s advisory committee meeting on eteplirsen saw a unified show of support from the patients and scientists who live with, study and treat Duchenne. The testimony from Duchenne experts during the open public hearing clearly underscores the positive data generated in the small clinical trial,” the co-founder of Race to Yes, Tracy Seckler of Charley’s Fund, said in a press release.

The Advisory Committee to the FDA, an independent panel of experts, met on April 25 to review eteplirsen. During the 12-hour meeting attended by more than 800 researchers, clinicians, and patients and their families from all over the world, compelling evidence in support of the efficacy and safety of the drug were presented.

However, the advisory committee voted against accelerated approval based on the fact that eteplirsen’s effect was too small and variable.

In clinical trials conducted over five years, eteplirsen has been shown to delay the progression of the disease based on the six-minute walk test, which measures the distance walked by trial participants in six minutes. But the trial only involved 12 boys.

The FDA will take into account the recommendations of the advisory committee, but is not obliged to follow them.

Under Food and Drug Administration Safety and Innovation Act (FDASIA), Congress gave the FDA the authority to grant accelerated approval for new treatments for life-threatening rare diseases such as Duchenne muscular dystrophy (DMD).

Marissa Penrod of Team Joseph, a co-founder of Race to Yes, said: “We encourage the FDA to honor FDASIA, and fully utilize the tools Congress has given them.”

A final FDA decision on whether to grant accelerated approval for eteplirsen, which is produced by Sarepta Therapeutics, is expected on May 26.

Eteplirsen is a drug designed to restore the reading frame of the dystrophin gene. In some Duchenne cases, a portion of this gene is missing, resulting in the remaining part not “fitting together” properly. By skipping over part of the remaining gene, the reading frame can be restored allowing the body to make a shorter but still functional dystrophin protein.

Race to Yes is a community-driven initiative that supports and campaigns for the rapid regulatory approval of DMD drugs.

Read our news story summarizing the approval process for eteplirsen: FDA Panel Casts Some Doubt on DMD Drug Eteplirsen’s Accelerated Approval.