US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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FDA Extends PDUFA Goal Date for Eteplirsen

Background: Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has extended the PDUFA goal date for eteplirsen by a standard extension period of three months. The new date by which the FDA must make a decision about whether to approve eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) is May 26, 2016.

The extension provides time for the FDA to complete a full review of eteplirsen. It follows Sarepta’s submission of new four-year clinical effectiveness data, which has been designated as a major amendment to the New Drug Application for eteplirsen.

Eteplirsen is an “exon-skipping” drug that targets a section of DNA called exon 51, and may help up to 13 percent of DMD patients. Exon skipping is a treatment strategy in which sections of genetic code are “skipped,” allowing cells to manufacture partially functional dystrophin, the muscle protein missing in DMD. Exon skipping is not a cure for DMD, but potentially could lessen the severe muscle weakness and atrophy that is the hallmark of the disease.

The rescheduled date for the advisory committee meeting to review eteplirsen has not yet been determined.

See below to read Sarepta’s press release:

Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen