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# FDA Priority Reviews for Marathon Duchenne NDAs
- URL: https://www.fdaweb.com/fda-priority-reviews-for-marathon-duchenne-ndas/
- Published: 2016-08-10T12:00:00.000Z
- Updated: 2026-09-14T21:23:29.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5136470

FDA has accepted for priority review two Marathon Pharmaceuticals NDAs for the investigational drug deflazacort — one for an immediate-release tablet formulation and the other for an oral suspension formulation — for treating patients with Duchenne muscular dystrophy (DMD). The submissions are expected to receive a user fee review target date of 2/2017.

The filings are supported by preclinical and clinical study data, including two pivotal clinical efficacy trials exclusively licensed by Marathon in more than 200 Duchenne patients aged five to 15 years. “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,” the company says. 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 U.S. and provides deflazacort to patients with Duchenne free of charge during the NDA review process.