ID: 2017-039 Novel Drug for Thalassemia & Hemochromatosis (Iron Overload)
Principal Investigator: Richard Watt
Thalassemias are inherited blood disorders characterized by abnormal hemoglobin production. Treatment depends on type and severity, with the more severe cases requiring regular blood transfusions or iron chelation. In 2013, an estimated 280 million patients worldwide suffered from some form of thalassemia, with 439,000 suffering from severe disease. Complications of thalassemia include excessive iron levels, heart or liver disease, infections and osteoporosis. Hemochromatosis (or iron overload) is a genetically inherited metabolic disease that causes the body to absorb excessive iron from the digestive tract, affecting an estimated one million people in the U.S. alone. Over time, this excess iron accumulates to unhealthy levels in tissues throughout the body. Symptoms of the disease typically do not appear until age 40 and include liver disease, weakness and lethargy, infections, joint pain, and reproductive disorders. Currently, the two most common treatments for hemochromatosis are therapeutic phlebotomy and chelation therapy. However, these treatments are costly and can result in unpleasant side effects. This invention embodies a novel hemochromatosis treatment that has the potential to serve as an alternative treatment for patients suffering from iron overload. In its unique use of a hepcidin inhibitor, this technology stabilizes the body’s natural iron secretion mechanisms from tissue and in combination with an iron chelator, can provide a safe and effective treatment option for hemochromatosis patients.
About the Market:
In terms of the thalassemia treatment market outlook, the WHO estimates that global hemoglobinopathies market will reach over $8.8 billion by 2020. Today, hemoglobin disorders such as thalassemia are endemic in over 60% of 229 countries. Over 70% of these countries’ births are affected by these disorders, with an estimated 85% mortality rate among infants below five years suffering from thalassemia. Current hemochromatosis treatment markets are projected to witness steady and promising growth over the next few year. The global phlebotomy treatments market is projected to be worth $9.9 billion by 2021, growing at a CAGR of 5.3%, and the global chelating agents market is expected to reach $6.8 billion by 2021, growing at a CAGR of 4.21%. Key market drivers are expanding global healthcare, especially in developing countries where many hemochromatosis sufferers have largely been left untreated. Lifestyle changes leading to increased stress levels may be linked to hemochromatosis-inducing mutations, and some believe that climate change may cause increased prevalence of this disease in the coming years. Currently, therapeutic phlebotomy is generally the preferred treatment by both patients and physicians because it is a simpler and possibly more effective therapy than chelation and causes fewer drug-induced side effects. However, as therapeutic phlebotomy entails uncomfortable long-term removal of blood from the body, patients and physicians will likely embrace future therapies in this market that offer patients more comfortable, yet safe and effective treatment options. Thus, licensees of this invention are poised to gain strategic competitive advantage in this market.
For more information, contact Mike Alder (801-422-3049)
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