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FDA Grants Orphan Designation to Mallinckrodt’s potential drug for Duchenne

Posted on: July 18th, 2017 | 0 comments

—-The U.S. Food and Drug Administation grants orphan designation to Mallinckrodt Pharmaceuticals‘ drug candidate MNK-1411 for treatment of Duchenne Muscular Dystrophy—-


MNK-1411 is an injection composed of a formulation of tetracosactide, which is a synthetic melanocortin receptor agonist. Melanocortin receptor agonists are hormones that activate melanocortin receptors and have been shown to have potential therapeutic effects on mouse models of Duchenne.

FDA’s orphan drug designation is a special status given to drugs that affect rare diseases, defined as having fewer than 200,000 patients in the United States. It can also be given for drugs that affect more than 200,000 people, but only if the drug company is not expected to make a profit by developing and marketing the drug. To earn the designation, a drug must provide more benefit than currently existing therapeutics.

MNK-1411 is currently approved for the treatment of some autoimmune and inflammatory diseases outside of the U.S., but has not yet been approved for use in the US.

A Phase 1 clinical trial has been conducted for MNK-1411 and Mallinckrodt is analysing the data obtained from that study to determine the optimal dose for a Phase 2 clinical trial set to begin in late 2017.

Prior to obtaining the orphan designation, the FDA also granted Fast Track designation to Mallinckrodt for MNK-1411 in August 2016. This designation allowed an expedited process for development of MNK-1411 and review by the FDA for the treatment of Duchenne.


“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,” Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt said 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.”


The original article came from Muscular Dystrophy News Today.


Action Duchenne will be following developments closely and look forward to sharing more information with you as we get it. As always the best place in the UK to get updates on all aspects of Duchenne is the annual Action Duchenne International Conference. This year the event is 10-12 November at the Hilton Birmingham Metropole.

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