Sangart's MP4CO receives orphan drug designation for acute pain in sickle cell disease

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced that its investigational biopharmaceutical product, MP4CO, has been awarded orphan drug designation by the U.S. Food and Drug Administration (FDA) for use in treating acute painful sickling crises in patients with sickle cell disease.

Orphan drug designation is awarded to drugs and biologics which are being developed for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US. This designation provides companies with financial incentives that can help support development, as well as market exclusivity if the compound is ultimately approved for sale by the FDA. As with all biopharmaceutical products, MP4CO will only receive FDA marketing approval if it is first shown to be safe and effective in human clinical trials, which have yet to be performed.

MP4CO is designed to deliver therapeutic levels of carbon monoxide (CO) to patients suffering from a sickle cell crisis. CO stabilizes hemoglobin S, an abnormal type of hemoglobin, and prevents sickling of red blood cells. The addition of MP4CO to existing treatment protocols may alleviate pain associated with a sickle cell crisis and potentially reduce the duration of a crisis. Clinically, this could mean preventing hospitalization or shorter hospital stays, reducing the need for addictive narcotic analgesics, and an improved quality of life for patients with sickle cell disease.

"This orphan drug designation is another significant milestone in the development of the MP4CO product," said Brian O'Callaghan, President and Chief Executive Officer of Sangart. "We look forward to working closely with the FDA on our clinical program and advancing this new treatment option into clinical studies."

Sickle cell disease is an inherited hemoglobin disorder that affects red blood cell circulation in millions of people around the world. People with sickle cell disease have red blood cells that contain mostly hemoglobin S, an abnormal type of hemoglobin. Sometimes these red blood cells become crescent or sickle-shaped and have difficulty passing through small blood vessels. This is known as a "sickle crisis". When sickle-shaped cells block small blood vessels, less blood circulates through the body, causing damage to tissues that do not receive a normal blood flow. There are currently no approved medications to treat sickle cell crises, and only symptomatic relief is available.