A recently established committee will discuss a novel therapeutic option for Duchenne muscular dystrophy (DMD), according to a press release made by Sarepta Therapeutics, Inc.

“It is our understanding that as one of the first gene therapy BLAs founded on a surrogate endpoint, the advisory committee will primarily relate to the totality of evidence supporting the conclusion that the SRP 9001 dystrophin is reasonably likely to predict clinical benefit, the standard for accelerated approval,” said Doug Ingram, president and chief executive officer of Sarepta.

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The decision the US Food and Drug Administration (FDA) announced at the most recent cycle meeting for the biologics license application of SRP-9001 will translate into the formation of an advisory committee that will take place on May 29 of this year.

The president of Sarepta explained that this action comes from the interest of the FDA in looking into the surrogate endpoints, approaches, and biomarkers for infrequent but potentially mortal and debilitating entities. This may lead to faster approval to advance genetic and cellular therapies that target such diseases.

Although this public committee represents a different direction than that of the initial thought process to achieve the approval of SRP-9001, the developers affirmed that they have been preparing for a meeting like this since 2022. The team behind this novel drug will present the evidence that supports the therapeutic potential of SRP-9001 in this meeting.

“While we are disappointed that we must communicate a change in decision after our prior statement on the topic, we are not disappointed with the decision to hold an advisory committee,” added Doud Ingram.

SRP-9001, also called delandistrogene moxeparvovec, is a gene transfer therapy currently undergoing experiments and testing and aims to introduce SRP-9001 in muscle tissue in order to promote the production of the dystrophin components. This treatment benefits patients with DMD and other muscular diseases such as limb-girdle muscular dystrophies (LGMDs).


Sarepta therapeutics announces advisory committee meeting will be held for SRP-9001. Sarepta Therapeutics Inc; Published online March 16, 2023.