Additional Relevant Details
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.1 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.