14 June 2016 - Deflazacort has fast track status, orphan drug designation and rare paediatric disease designation for Duchenne muscular dystrophy.

Marathon Pharmaceuticals today announced it has submitted a New Drug Application (NDA) to the U.S. FDA for the investigational drug deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.

“This NDA submission starts a process that we hope will result in broad access to this medication for all of those living with Duchenne who need it,” said Jeff Aronin, Chief Executive Officer, Marathon Pharmaceuticals. “We recognize the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application.”

The NDA filing is supported by a full preclinical and clinical study program, including two pivotal clinical efficacy trials exclusively licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of age. These data show that deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology and in one of the studies ambulation status. Marathon additionally conducted seven clinical pharmacology and safety studies of deflazacort and nine preclinical studies to support either the initiation of clinical studies or marketing approval. An expanded access program, Access DMD, is ongoing in the United States and provides deflazacort to patients with Duchenne free of charge during the NDA review process.

For more details, go to: http://marathonpharma.com/news/2016/06/marathon-pharmaceuticals-announces-submission-deflazacort-new-drug-application-fda/