Rare Disease Reports: FDA Accepts Sarepta’s Sarepta’s NDA for Eteplirsen. PDUFA Date Set

September 17, 2015

Thanks to Rare Disease Report for this post.

Sarepta Therapeutics announced that the FDA has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.

According to Sarepta, the FDA has completed its filing review and has determined that the application is sufficiently complete to permit a substantive review.

The Prescription Drug User Fee Act (PDUFA) action date is February 26, 2016.

Edward M. Kaye, interim chief executive officer and chief medical officer of Sarepta stated,“We are pleased with the FDA’s acceptance of our NDA for eteplirsen, as it represents an important milestone, not only for Sarepta, but for the Duchenne community. We look forward to continuing to work closely with the FDA during the regulatory review process.”

Dr. Kaye added, “We believe eteplirsen has the potential to make a meaningful impact on the lives of patients amenable to skipping exon 51 and we aim to build on our experience with eteplirsen to work with the FDA to inform and potentially expedite the clinical and regulatory pathway for the follow on exons, with the goal of reaching as many patients amenable to exon skipping as possible.”

Last week, BioMarin and Sarepta were given pediatric rare disease designations form the FDA for their two drugs that are aimed at the same subset of Duchenne muscular dystrophy boys (those amenable to exon 51 skipping).  BioMarin’s NDA was accepted in June with a PDUFA date set at December 27, 2015.  Whether or not the FDA sticks to that schedule is not known. There is tremendous pressure from the Duchenne patient community to approve both drugs and to approve them sooner rather than later.

In a recent interview with Rare Disease Report, former president and CEO of Sarepta, Chris Garabedian stated he thought the FDA would accept the NDA. And he believes the drug will be approved. Click here to see a clip from that interview.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with DMD lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

There are an estimated 35,000 patients with DMD in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.

There are currently no treatments for these patients but several drugs are in late stage clinical development, including Sarepta’s eteplirsen and BioMarin’sdrisapersen which should be effective in the same 13% of the Duchenne population who would benefit from exon 51 skipping therapy (i.e., those with mutations near exon 51 of the dystrophin gene). Both eteplirsen and drisapersen have now completed their rolling NDA submissions. Other drugs being investigated for DMD include PTC Therapeutics’ ataluren which should be effective in another 13% subset who have nonsense mutations in the dystrophin gene and Lilly’s tadalafil being targeted for all patients with DMD.


Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping [news release]. Cambridge, MA: Sarepta Therapeutics; August 25, 2015.]

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