Mallinckrodt, a specialty pharmaceutical company, announced the FDA has granted orphan drug designation to MNK-1411, a long-acting formulation containing cosyntropin acetate under investigation for the treatment of Duchenne muscular dystrophy (DMD).
"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. "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."
The FDA's Orphan Drug Designation program provides orphan status to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. The designation provides Mallinckrodt marketing exclusivity in the U.S. for DMD for a seven-year period following FDA approval, as well as the ability to apply for research funding, tax credits related to certain research costs, and a waiver of the FDA application user fee.
Mallinckrodt has completed a phase I study for MNK-1411 in healthy volunteers, and the company is using the information that was derived to determine optimal dosing for patients in the phase II trial, which should begin later this year.
In August 2016, the FDA granted the company's request for a Fast Track designation for its Investigational New Drug application for MNK-1411 in the treatment of DMD. The Fast Track designation is a process designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need.