La Jolla Pharmaceutical Company Receives Positive Opinion from European Orphan Committee for LJPC-401

La Jolla Pharmaceutical Company

(the "Company" or "La Jolla"), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced 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 anemia and often require frequent and life-long blood transfusions. These blood transfusions, while necessary to treat anemia, 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 hemorrhage. 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. La Jolla is developing LJPC-401 for the treatment of iron overload, which occurs as a result of diseases such as HH, beta thalassemia and sickle cell disease. In a separate decision, the EMA's Innovation Task Force (ITF) has granted a meeting with La Jolla to review future development plans for LJPC-401. The ITF provides an interactive platform for applicants with novel and innovative therapies to proactively discuss the scientific, legal and regulatory aspects of development for such therapies with the EMA. This early dialogue is intended to contribute to the preparedness of an applicant and to increase EMA awareness and education of emerging therapies and technologies. "We believe that LJPC-401 can have a major impact on the lives of patients suffering from chronic iron overload," said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. "We are encouraged by the positive feedback and support of the EU regulatory authorities and are excited to further our clinical development of LJPC-401." Emas Pharma Limited served as La Jolla's European regulatory representative for these regulatory interactions.