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Press Release

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

La Jolla Pharmaceutical
Posted on: 25 Oct 16


La Jolla Pharmaceutical Company (LJPC) (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 (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.”

About European Orphan Drug Designation

Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status in the European Union (EU), the medicine must be for the treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the EU and for which no satisfactory treatments exist or, where they do exist, the medicine will be of significant benefit to those affected by that condition.

Applications for orphan designation are evaluated by the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), which provides its opinion on whether or not the medicine qualifies as an orphan medicine for the treatment, prevention or diagnosis of a rare disease. If the COMP issues a positive opinion, the European Commission (EC) may then grant the medicine orphan status.

An orphan designation allows a pharmaceutical company to benefit from incentives from the EU to develop a medicine for a rare disease, such as reduced fees, regulatory support during the product development phase, access to the centralized authorization procedure (a single application for all EU countries), and 10 years of market exclusivity once the medicine is approved.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of synthetic human hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death.

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Editor's Details

Mike Wood

Last updated on: 25/10/2016

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