U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATI-1013, a fully human, anti-nicotine monoclonal antibody for the treatment of Buerger’s Disease.
Buerger’s Disease (thromboangitis obliterans) is a rare disorder that is caused by cigarette smoking or ingesting nicotine from other sources (such as smokeless tobacco) in which small blood vessels in the arms and or legs become blocked. This condition reduces blood flow to the affected areas, causing pain, skin ulcers and eventual severe tissue damage that often leads to gangrene and the need for amputation. Buerger’s Disease largely affects young or middle-aged cigarette smokers.
“The mechanism by which smoking induces Buerger’s disease is not known, but nicotine plays the most important role in initiation and progression of this vascular disease,” says Jeffrey W. Olin, DO, FACC, FAHA, Professor of Medicine at Icahn School of Medicine at Mount Sinai Hospital. “While some patients are able to stop smoking or using tobacco in other forms, many are not, therefore a human monoclonal antibody that blocks the effects of nicotine may be a major advance in the treatment of patients with Buerger’s disease.”
ATI-1013 is a proprietary compound that neutralizes nicotine in the blood. Studies in animals have shown that following an injection of nicotine, ATI-1013 blocks nicotine-induced increases in blood pressure, reduces nicotine levels in the brain by more than 90%, and reduces the vasoconstrictive peripheral effects of nicotine on blood vessels. Research into ATI-1013 has been partially funded by a grant from the National Institute of Health, National Institute on Drug Abuse.2
“Currently there are no approved treatments for Buerger’s Disease, which is a distinct and very severe type of peripheral arterial disease (PAD),” says Matthew Kalnik, Ph.D., President and Chief Executive Officer, Antidote Therapeutics, Inc. “This is an important milestone and we’re very pleased to receive Orphan Drug Designation from the FDA.”
The Orphan Drug Act grants orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S. Under the Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA.
