CAMBRIDGE, Mass.–(BUSINESS WIRE)–Feb. 8, 2016– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) date for eteplirsen has been extended to May 26, 2016. The rescheduled date for the Peripheral and Central Nervous System Advisory Committee meeting has not yet been determined.
The FDA notified Sarepta that its January 8, 2016 submission of 4-year clinical effectiveness data, which included additional six minute walk test (6MWT) and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. TheFDA stated that the PDUFA goal date has been extended by three months to allow for a full review of the submission. As described in the Sarepta Advisory Committee Briefing Document Addendum, the principal basis for establishing the effectiveness of eteplirsen is a comparison of patients in Study 201/202 to a historical control group.
“While our primary goal is to bring treatment to patients with Duchenne as quickly as possible, we appreciate the efforts of the FDA to conduct a complete review of all of the data supporting our NDA and we remain committed to working closely with them throughout the remainder of the regulatory process”, said Edward Kaye, Sarepta’s interim chief executive officer and chief medical officer.
The FDA has previously granted eteplirsen Priority Review status, which is designated for drugs that provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to eteplirsen, as well Orphan Drug Designation and Fast Track Status.
It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13 percent of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
About Sarepta Therapeutics
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of rare, infectious and other diseases. For more information, please visit us at www.sarepta.com.
Eteplirsen is designed to address the underlying cause of DMD by restoring the dystrophin messenger RNA (mRNA) reading frame, thus enabling the production of a shorter, functional form of the dystrophin protein. Eteplirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Approximately 13 percent of the DMD population is amenable to exon 51 skipping. Data from clinical studies of eteplirsen in DMD patients have demonstrated a consistent safety and tolerability profile and have also shown measurable dystrophin protein expression. Promoting the synthesis of a shorter dystrophin protein is intended to slow the decline of ambulation and mobility seen in DMD patients. There currently is no approved treatment in the United States for DMD and eteplirsen has not been approved by the FDA or any regulatory authority for the treatment of DMD.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare 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 3,500-5,000 boys worldwide. A devastating and 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.