Mallinckrodt’s Duchenne MD Therapy IND Application Granted FDA Fast Track Designation

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Mallinckrodt’s investigational new drug (IND) application for Synacthen Depot to treat Duchenne muscular dystrophy (DMD).

The invesigational drug is a depot formulation (a sustained-action drug formulation that allows slow release and gradual absorption) of Synacthen (tetracosactide), a synthetic 24 amino acid melanocortin receptor agonist.

The formulation is typically used for short-term therapy in conditions for which glucocorticoids are indicated in principle – for instance, in ulcerative colitis and Crohn’s disease, juvenile rheumatoid arthritis, or as adjunct therapy in patients with rheumatoid arthritis and osteoarthrosis. Synacthen Depot may be particularly useful in patients unable to tolerate oral glucocorticoid therapy or in patients where normal therapeutic doses of glucocorticoids have been ineffective.

“We are pleased with the FDA’s determination that our IND application for use of Synacthen Depot in treatment of Duchenne muscular dystrophy meets the agency’s established criteria for a Fast Track designation, and are happy to report the first patients have been dosed in the initial Phase 1 trial,” Dr. Steven Romano, MD, Mallinckrodt senior vice president and chief scientific officer, said in a press release.

“DMD is a devastating disease for which there are few effective treatment options, and Mallinckrodt believes Synacthen Depot may have potential to offer physicians and patients a new alternative,” he said.

The protein dystrophin helps keep muscle cells intact. DMD is a genetic disorder caused by an absence of dystrophin, characterized by progressive muscle degeneration and weakness. It is one of nine different types of muscular dystrophy.

An absence of dystrophin leads to muscle weakness, first affecting the muscles of the hips, pelvic area, thighs, and shoulders, and later the skeletal (voluntary) muscles in the arms, legs, and torso. The onset of symptoms begins in early childhood, usually between the ages of 3 and 5, and primarily affects boys.

Synacthen Depot is approved and marketed outside the United States for certain autoimmune and inflammatory conditions, but has never been approved for use in U.S. patients.

Fast Track designations are designed to facilitate the development and expedite the review of drugs meant to treat serious conditions and fill an unmet medical need. The purpose is to get important new therapies to the patient as quickly as possible.