Background: On Monday, June 29, BioMarin announced that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen. Both “exon-skipping” drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients. Exon skipping is a treatment strategy in which sections of genetic code are “skipped,” allowing the creation of 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 this disease.
The FDA has accepted BioMarin’s drisapersen for priority review, and Sarepta has formally requested a priority review for eteplirsen. Both companies hope to have an FDA decision by early 2016.
Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.:
“Today marks a huge step forward for our Duchenne families as two potential therapies formally move toward FDA review. We are as close as we’ve ever been to having approved therapies for one of the most common and debilitating forms of muscular dystrophy.
“MDA has provided support for the development of exon skipping since the 1990s and we are proud to have funded foundational research that has made these new treatments possible. It is a sign of great progress to witness multiple companies working on multiple therapies to urgently get treatments to our families.
“MDA is committed to bringing safe and effective treatments and cures to children and adults living with life-threatening neuromuscular diseases as quickly as possible. Today, there are new treatments in the pipeline that were inconceivable just a few years ago and more potential treatments are expected during the next five years than in the past five decades. As these treatments enter the regulatory review phase, MDA continues to work hand-in-hand with families, health care professionals, scientists and the FDA to ensure it has the necessary information and support to efficiently make decisions on potential life-changing drugs and to move these treatments from the labs to our families’ living rooms.”
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.