Earlier today, it was announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Mallinckrodt plc for their potential Duchenne muscular dystrophy (DMD) treatment, MNK-1411.
MNK-1411 is a long-acting depot formulation of tetracosactide, a melanocortin receptor agonist.
DMD is the result of a lack of functional dystrophin protein, which assists in keeping muscles intact. Patients with this progressive muscle condition have been known to lose walking abilities at as early as age 10, and experience potentially fatal lung and heart impediments into their late teenage years and their early 20s.
"We are pleased the FDA has granted orphan drug status to MNK-1411 for potential treatment of DMD, a serious disease for which there are limited treatment options," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer in a press release
"Mallinckrodt believes MNK-1411 may have potential to offer physicians and patients a new treatment alternative, and this designation is an important step forward for the development program. We continue to advance our broad clinical and regulatory strategy in areas of high unmet medical need."
MNK-1411 has completed a Phase 1 study in healthy volunteers, and in August 2016, the FDA granted a Fast Track designation for its Investigational New Drug application for the treatment of DMD. The drug is currently approved and marketed outside of the U.S. for an array of autoimmune and inflammatory conditions, but was never granted approval for the use U.S. patients.
Using the information derived from the Phase 1 study to determine the ideal dosage for patients, Mallinckrodt has plans to begin the Phase 2 trial in the fourth quarter of 2017.
For more information on FDA applications, designations and approvals, follow Rare Disease Report