La Jolla Pharmaceutical (LJPC) Announces Receipt of Positive EMA CHMP for LJPC-401 in SCD

October 24, 2016 8:10 AM EDT
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La Jolla Pharmaceutical Company (Nasdaq: LJPC) announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending LJPC-401 (synthetic human hepcidin) for designation as an orphan medicinal product for the treatment of Sickle Cell Disease (SCD).

SCD is the most common inherited blood disorder in the United States and is caused by a genetic mutation that results in the production of abnormal hemoglobin, the body’s natural oxygen-carrying molecule contained in red blood cells. The abnormal hemoglobin causes the red blood cells to form a “sickle,” or crescent, shape, which may cause occlusion of blood vessels. Patients with severe forms suffer from sometimes life-threatening chronic hemolytic anemia, strokes, and damage to vital organs such as the lungs, spleen, kidney and liver. In patients with chronic hemolytic anemia, hepcidin levels may also be suppressed, which may lead to iron overload. Standard treatment of SCD includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause excess iron accumulation, which in turn is toxic to vital organs, such as the liver and heart. The only currently approved treatments for iron overload are iron chelators, which may cause kidney failure, liver failure or gastrointestinal hemorrhage. In addition to potentially improving iron overload in these patients, LJPC-401 holds the potential to improve the consequences of the disease by reducing the red cell hemoglobin concentration leading to less sickle cell formation.

LJPC-401 is La Jolla’s novel formulation of synthetic human hepcidin, a naturally occurring peptide hormone that is the body’s regulator of iron absorption and distribution. Hepcidin prevents abnormal 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 potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis, beta thalassemia, SCD and myelodysplastic syndrome. In September 2015, the COMP designated LJPC-401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major.

“We are encouraged by the positive feedback and continued support of the European regulatory authorities,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “Following our recently reported, positive results from our Phase 1 study of LJPC-401, which demonstrated a clear, dose-dependent effect of LJPC-401 on serum iron, and our reaching of an agreement with the EMA on study design, we look forward to initiating our pivotal study of LJPC-401 in mid-2017.”

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