FDA Continues Review of Controversial DMD Treatment

The FDA is continuing to review the NDA past the PDUFA goal date
The FDA is continuing to review the NDA past the PDUFA goal date

Sarepta announced that the Food and Drug Administration (FDA) is continuing the pending New Drug Application (NDA) review for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

The FDA has also notified the company that they will not be able to complete the review by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. The FDA will continue to review the NDA past the PDUFA goal date and strive to complete their work in as timely a manner as possible. 

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Eteplirsen is an investigational therapy that utilizes Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13% of the total DMD population.

Last month,  a majority of FDA's Advisory Committee voted against recommending eteplirsen. With no current treatment for DMD, patients, parents, and clinicians have been lobbying the FDA for years to gain approval for the treatment.

For more information call (855) 363–7547 or visit Sarepta.com.

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