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FDA Accepts Application for Muscular Dystrophy Drug Eteplirsen

Approval decision set for February 2016

A new drug application (NDA) for eteplirsen (Sarepta Therapeutics) has been submitted to the FDA for the potential treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

The FDA has completed its filing review and has determined that the NDA is sufficiently complete to permit a substantive review. The Prescription Drug User Fee Act (PDUFA) action date for a decision on the application is February 26, 2015. The FDA has granted “priority review” status to eteplirsen. This designation is given to drugs that offer a clinical benefit over existing therapies, or that provide a treatment where no adequate therapy exists.

Eteplirsen was designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated the restoration of dystrophin protein expression.

Eteplirsen uses phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene, thereby enabling the repair of specific genetic mutations that affect approximately 13% of the total DMD population. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, but still functional, form of dystrophin from messenger RNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and to prolong and improve the quality of life for patients with DMD.

DMD is a rare, X-linked degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys born worldwide. An incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle-fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck, and other areas. Eventually, increasing difficulty in breathing due to respiratory-muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.

Source: Sarepta Therapeutics; August 25, 2015.

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