FDA Grants Orphan Drug Designation to Mallinckrodt Development Product for Potential Treatment of Duchenne Muscular Dystrophy

STAINES-UPON-THAMES, United Kingdom, July 12, 2017 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading specialty pharmaceutical company, today announced the U.S. Food and Drug Administration (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 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.

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.

About MNK-1411 (Cosyntropin Injection)
MNK-1411 (cosyntropin injection) is a depot formulation of tetracosactide, a synthetic 24 amino acid melanocortin receptor agonist. The product is approved and marketed outside of the U.S. for certain autoimmune and inflammatory conditions, but has never been approved for use in patients within the U.S.

About Duchenne Muscular Dystrophy (DMD)
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disease primarily affects boys. Symptoms include muscle weakness, which can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs and shoulders, and later the skeletal (voluntary) muscles in the arms, legs and trunk.¹

ABOUT MALLINCKRODT

Mallinckrodt is a global business that develops, manufactures, markets and distributes specialty pharmaceutical products and therapies. Areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; and analgesics and hemostasis products. The company's core strengths include the acquisition and management of highly regulated raw materials and specialized chemistry, formulation and manufacturing capabilities. The company's Specialty Brands segment includes branded medicines and its Specialty Generics segment includes specialty generic drugs, active pharmaceutical ingredients and external manufacturing. To learn more about Mallinckrodt, visit www.mallinckrodt.com.

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¹ https://www.mda.org/disease/duchenne-muscular-dystrophy (Accessed Aug. 21, 2016)

SOURCE Mallinckrodt plc