US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

About Us

Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

PTC Therapeutics Completes NDA for Ataluren to Treat DMD

Background: PTC Therapeutics today announced that it has completed submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval to market ataluren (Translarna) to treat Duchenne muscular dystrophy (DMD) in the United States.

The South Plainfield, N.J.-based company also said it has submitted the results of its phase 3 ACT DMD clinical trial to the European Medicines Agency (EMA) in connection with its approval of the drug in the European Union in August 2014.

Ataluren, a protein restoration therapy for the treatment of nonsense mutation DMD, acts by changing the way muscle cells interpret genetic information, coaxing them to produce a needed muscle protein called "dystrophin" despite the presence of a mutation in the dystrophin gene. The drug, which is taken by mouth, targets a particular type of genetic mutation known as a "nonsense mutation" or "premature stop codon," which is present in about 13 percent of DMD patients. This type of mutation results in the making of a protein that is not complete and is therefore not functional.

MDA has contributed nearly $3 million towards ataluren’s development and phase 2 clinical testing.

Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.:

“With every passing day, we get closer to the time when safe and effective treatment options will be available for kids and adults living with DMD. We remain hopeful that time is near, as ataluren joins a handful of other drugs passing through late-stage trials and moving through the FDA review process.

“MDA supported foundational work in this space, and contributed funding to ataluren's early-stage development and clinical testing. We are gratified to see all that effort come to fruition and optimistic that this treatment has reached the final stages that could soon result in it being approved and made available to the DMD community.

“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.”

About MDA

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.

Visit and follow us at and @MDAnews. Learn more about MDA's mission by watching this video.