Marathon Pharmaceuticals submits deflazacort NDA to treat Duchenne muscular dystrophy

Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. It has fast track status, orphan drug designation and rare pediatric disease designation for DMD.

One of the pivotal studies demonstrated that treatment with deflazacort enhanced muscle strength versus placebo at 12 weeks.

The NDA submission was based on a preclinical and clinical study program, including two Marathon-licensed clinical efficacy trials in over 200 Duchenne patients aged 5 to 15 years.

Marathon said deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology and in one of the trials ambulation status.

The company also carried out seven clinical pharmacology and safety studies of deflazacort and nine preclinical trials.

Marathon Pharmaceuticals CEO Jeff Aronin said: "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.

"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."

Deflazacort is not currently approved in the US for any indication. Versions of the drug are available in various countries outside the US where it is approved for several indications, but not for Duchenne.

Image: Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. Photo: courtesy of m_bartosch/FreeDigitalPhotos.net.