Drugs in development

As a result of considerable and ongoing investments in clinical research there are new pharmaceutical treatments in development to improve the care and quality of life of those living with Duchenne. Most promising are gene-based therapies that can express the missing dystrophin protein (exon skipping or mutation suppression).

The following two new treatments are either recently approved (in the case of Exondys 51 in the U.S), currently in late-stage development and going through regulatory review in some countries.

Exondys 51(TM) (Eteplirsen)  Recently approved by the FDA in the U.S., Exondys 51 is a drug developed by Sarepta Therapeutics, designed to skip exon 51.  (Note: Stand for Duchenne Canada is attempting to get additional information on plans for access to this treatment in Canada.  If you would like to receive updates, please contact us)

A candidate for exon 53 skipping (SRP-4053) is also in clinical development, and a candidate for exon 45 skipping (SRP-4045) has entered early clinical development. Sarepta also has other drug candidates in discovery and preclinical development that are designed to skip exons 44, 52, 50, 43, 55, 8 and 35.  Read more

Translarna™ (ataluren) discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States and Canada. Read more

BioMarin Pharmaceutical Inc. announced on May 31, 2016 that it was discontinuing the development of Kyndrisa™ (drisapersen) for Duchenne muscular dystrophy, along with three related products. Read more

To see a video of Dr. Jean Mah, Associate Professor of Pediatric Neurology at Alberta Children’s Hospital speak on emerging therapies for DMD, click here.