Orphan diseases are defined by the U.S. Food and Drug Administration as diseases affecting less than 200,000 people in the United States. Conditions that fall under the umbrella of orphan diseases include Huntington’s disease, Lou Gehrig’s disease, and muscular dystrophy. Although these conditions may not affect as many people as diseases such as lung or breast cancer, and may therefore generate comparatively smaller drug sales, a significant market opportunity exists for companies willing to venture into the arena of orphan diseases. The U.S. Food and Drug Administration, through the Orphan Drug Act of 1983, offers tax and marketing incentives to companies developing drugs for rare diseases. And according to a new report from business information provider Bharatbook, the market for orphan drugs is expected to increase from an estimated $85 billion in 2009 to more than $112 billion in 2014, for a compound annual growth rate (CAGR) of 5.7%.
AesRx is one company that could benefit from the growing market for orphan drugs. The Newton, Mass.-based biopharmaceutical company is developing a small-molecule drug for sickle cell disease, an inherited disorder that affects 13 million people worldwide (75,000 in the U.S.) according to company-provided statistics. Sickle cell disease causes red blood cells to become stiff and crescent-shaped. The affected cells may block capillaries and other small blood vessels, which can lead to a wide range of health complications such as chronic hemolytic anemia, acute chest syndrome, jaundice, pain, stroke, and cumulative tissue and organ damage. According to the Sickle Cell Disease Association of America, blood transfusions may help reduce pain episodes and the risk of complications, but patients who receive repeated transfusions may experience a toxic buildup of iron in the body. There is currently no adequate approved treatment for the disease. AesRx is developing a pill form of Aes-103, a small molecule that could reduce the number of red blood cells that can sickle. The company plans to begin human clinical trials in 2011.
The company is also developing Aes-210 to treat inflammatory diseases of the lower intestine, including distal ulcerative colitis, pouchitis and radiation induced proctitis. AesRx estimates that 400,000 to 500,000 people worldwide suffer from these diseases. Aes-210 has been granted Orphan Drug Status by the FDA. The drug is currently undergoing a Phase II clinical trial in patients with pouchitis, inflammation of an internal pouch created in patients who have had all or part of the colon removed to treat ulcerative colitis. The company reports that initial results have been encouraging.
AesRx is scheduled to present at the OneMedForum San Francisco 2011 in January. To learn more about the conference, which features promising emerging growth companies from the healthcare industry, please visit http://www.onemedplace.com/forum.
