FDA Committee will review Sarepta’s NDA for eteplirsen

Sarepta Therapeutics recently announced that its New Drug application (NDA) for eteplirsen will be reviewed by the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System (PCNS) Drug Advisory Committee on April 25.

In addition, May 26 is the Prescription Drug User Fee Act (PDUFA) action date for completion of the review conducted by the FDA.

Eteplirsen was developed to treat the primary source of Duchenne muscular dystrophy (DMD) by restoring the dystrophin messenger RNA (mRNA) reading frame. Restoring the mRNA will result in the production of a shorter, functional form of the dystrophin protein. The drug was granted Priority Review by the FDA. The status is given to potential medicines that provide treatment when no therapy exists. Eteplirsen was also granted Rare Pediatric Disease Designation, Orphan Drug Designation and Fast Track Status by the FDA.

Estimations suggest that DMD affects roughly one in every 3,500 to 5,000 boys on a global scale. Of those affected, 13 percent have mutations that can be addressed by eteplirsen.