Sangart, Inc. Receives European Orphan Drug Designation for the Use of MP4CO in Patients With Sickle Cell Disease
SAN DIEGO, Dec. 9 /PRNewswire/ -- 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 the European Commission (EC) has granted orphan medicinal product designation for MP4CO for the treatment of sickle cell disease. This designation provides ten years of market exclusivity in the European Union (EU) following marketing authorization.
MP4CO is designed to deliver therapeutic levels of carbon monoxide (CO) to patients suffering from a sickle cell crisis. CO stabilizes hemoglobin S 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, reducing the need for addictive narcotic analgesics, less time in the hospital if admitted, and an improved quality of life for patients with sickle cell disease.
The EC regulation for orphan medicinal products is designed to promote the development of drugs for rare diseases. Orphan medicinal product designation can confer numerous benefits to companies developing such treatments, including regulatory assistance, reduced regulatory fees associated with applying for marketing approval, and assistance with clinical trial design.
"Receiving orphan drug designation is an important step in our clinical development and planned commercialization of MP4CO," said Brian O'Callaghan, President and Chief Executive Officer of Sangart. "We are pleased to have reached this important milestone and look forward to working with the EMEA (European Agency for the Evaluation of Medicinal Products) to plan our clinical program and initiate our clinical studies. We remain committed to bringing a new therapeutic option to patients suffering from sickle cell crisis."
Sangart's product platform is based on the MP4 molecule, an investigational biopharmaceutical product designed to enhance the perfusion of oxygen-deprived (ischemic) tissues and provide targeted oxygen delivery in the capillaries. Carbon monoxide (CO) at very low doses stabilizes hemoglobin S and acts as a messenger to cells, reducing inflammation, reducing oxygen requirements and preventing programmed cell death. The MP4 molecule can be modified to carry CO and other gases to enhance therapeutic benefit for certain patients. MP4CO is designed to deliver therapeutic, non-toxic levels of CO providing an immediate metabolic signal to cells and reducing inflammation. Once the CO is released from the compound, the MP4 molecule is oxygenated in the lung and then delivers oxygen to the ischemic tissues.
MP4CO is made in the same manner as MP4OX, with the only difference being that a CO molecule is attached in place of oxygen in order to provide an additional therapeutic benefit to the patient.
About Sickle Cell Disease
Sickle cell disease is a genetic disorder that affects red blood cell circulation in millions of people around the world, including approximately 70,000 people in the United States and between 40,000 and 50,000 people in the European Union.
Sickle cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are disk shaped and can deform and move through small blood vessels to carry oxygen to all parts of the body. In sickle cell disease, as red blood cells circulate and oxygen is released in the circulatory system, the deoxygenated abnormal hemoglobin S polymerizes. When this occurs, the red blood cells can become sticky and elongated to look like a C-shaped farm tool called a 'sickle'. These sickled red blood cells do not mold to the size of the blood vessel. They obstruct small blood vessels and can block normal red blood cells from traveling through the circulatory system limiting oxygen delivery to tissues and organs. This is known as a "sickle crisis". There are currently no approved medications to treat sickle cell crises, and only symptomatic relief is available.
The number of crises a sickle cell disease patient experiences varies. Most sickle cell disease patients experience at least one sickle crisis at some point during their lives; some may suffer 15 or more crises each year. Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death.
Sangart is 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 delivery. Based on more than a decade of research, Sangart has refined the pegylation of human hemoglobin to create a molecule, MP4, with the ability to carry oxygen through the circulatory system to prevent and treat ischemia.
To learn more about Sangart, please visit the company's website at www.sangart.com.
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SOURCE Sangart, Inc.