The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Mallinckrodt for their potential Duchenne muscular dystrophy (DMD) treatment, MNK-1411 (a long-acting depot formulation of tetracosactide, a melanocortin receptor agonist).
DMD is a devastating and debilitating disease that is the result of a lack of functional dystrophin protein, which helps 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 issues into their late teenage years and their early 20s. DMD represents a market with significant unmet medical need. In September 2016, Sarepta Therapeutics gained accelerated approval for Exondys 51 in the U.S. for the treatment of patients suffering from DMD amenable to exon 51 skipping. Exondys 51 is the first approved disease-modifying therapy for DMD in the U.S. as well as the company’s first product to receive marketing approval.
“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.”
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 1 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 2 trial, which should begin later this year.