European Orphan Committee issues positive opinion for La Jolla's LJPC-401.
M2 EQUITYBITES-September 14, 2015-European Orphan Committee issues positive opinion for La Jolla's LJPC-401
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La Jolla Pharmaceutical Company (Nasdaq: LJPC), a US-based pharmaceutical company, announced on Friday that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending LJPC-401, La Jolla's novel formulation of hepcidin, for designation as an orphan medicinal product for the treatment of chronic iron overload requiring chelation therapy.
Chronic iron overload occurs in patients suffering from beta thalassemia, sickle cell disease and hereditary hemochromatosis (HH). The final opinion, which is subject to review and approval by the European Commission (EC), may include all or a subset of these conditions.
Beta thalassemia and sickle cell disease are genetic diseases of blood cells that can cause life-threatening anaemia and often require frequent and life-long blood transfusions. These blood transfusions, while necessary to treat anaemia, cause excessive iron accumulation in the body, which is toxic to vital organs. HH is a disease caused by a genetic deficiency in hepcidin production, resulting in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.
Iron chelators are drugs that bind to and help clear excessive iron from the body. However, chelators cause significant toxicity, including kidney failure, liver failure or gastrointestinal haemorrhage. LJPC-401 is La Jolla's novel formulation of hepcidin, a naturally occurring peptide hormone that is the body's regulator of iron absorption and distribution. Hepcidin prevents excessive iron accumulation in organs, such as the liver and heart, where it can cause significant damage and even result in death.
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