July 24, 2013
Sarepta Therapeutics Inc. today announced that it plans to apply for U.S. regulatory approval for eteplirsen, its rare disease drug intended to treat Duchenne muscular dystrophy, in the first half of 2014. If approved, this would be the first drug designed specifically for a muscular dystrophy. The patient community is watching the development of eteplirsen closely for several reasons. Individuals and families of those who may be able to use eteplirsen see this as a drug with true potential to slow the course of the disease, and are excited to see the possibility of the drug getting to market. Those with different mutations, unable to be treated with eteplirsen, are watching closely to see what the path forward will be for subsequent drugs — the sooner eteplirsen is on the market, the more resources Sarepta and other companies will have to devote to the next group of mutations in line. Sarepta has several additional drugs in development.
The following quote may be attributed to Valerie Cwik, M.D., MDA Executive Vice President of Research, and Chief Medical and Scientific Officer:
“Today’s announcement by Sarepta is another encouraging sign that new lifesaving treatments are on the horizon. MDA believes this is a remarkably hopeful time in the race to unlock new therapies, given decades of investments we have made in neuromuscular disease research, including $4 million in early-stage exon-skipping projects that led to the development of eteplirsen. On behalf of the families we serve, MDA remains committed to doing all it can to ensure that safe, effective drugs and therapies are introduced to the market as quickly as possible.”