Neurology Central

New evidence pushes Duchenne Muscular Dystrophy drug potentially closer to approval

Rare disease specialists, Marathon Pharmaceuticals, have announced conclusions from a Phase III study of the investigational glucocorticoid, termed deflazacort, for the treatment of Duchenne Muscular Dystrophy (DMD). The results were presented at the American Academy of Neurology (AAN) 68th Annual Meeting (15 – 21st April, Vancouver, BC, Canada).

The trial was a randomized, double-blind placebo controlled and active comparator study that involved 196 patients. All patients received either deflazacort 0.9 mg/kg/day, deflazacort 1.2 mg/kg/day, prednisone 0.75 mg/kg/day or placebo for 12 weeks.

Previously presented primary endpoints of the study indicated that deflazacort improved muscle strength in DMD patients in comparison to placebo at 12 weeks.

A further secondary endpoint, presented at AAN, highlights the results of timed functional tests (TFT), demonstrating a significant improvement in the ability to perform three tests (time from lying down to standing, time to climb four stairs, and time to run or walk 30 feet) in deflazacort patients from baseline to week 12 versus placebo.

In addition, post-hoc analyses indicate an effect of deflazacort on muscle strength and pulmonary function in DMD patients who had lost the ability to walk.

Currently there are no FDA approved treatments for DMD and following on from the positive results of deflazacort, the drug has been granted Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation.

Nancy Kuntz (Ann & Robert H. Lurie Children’s Hospital, IL, USA) commented: “As a physician who works with dozens of boys and young men with DMD, every day I see the impact this serious disease has on the patients and the families that care for them.”

Marathon Pharmaceuticals now hope to submit a New Drug Application for deflazacort in May this year, meaning, if approved, the drug may be available within the USA by early 2017.

Source: Marathon Pharmaceuticals press release