Searching for a cure, Improving lives

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.

Leave a Reply

Your email address will not be published. Required fields are marked *

Donate

Support Action Duchenne with a monthly or one off donation

Donate

Events

View our events

View All Events

Registry

Join the DMD registry for access to clinical trials

Register

Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

November 17th, 2017

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is

Tell me more

Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

July 26th, 2017

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function

Tell me more

FDA new approach would reduce number of patients treated with placebo

December 11th, 2017

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach

Tell me more

Results announced from survey ‘Juggling care and daily life’

December 11th, 2017

The 17th October 2017 was an important date for the rare disease community.  The highlights of the

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

wordpress counter

Stay informed with regular updates from Action Duchenne