La Jolla Pharma Receives Positive Opinion From European Orphan Committee For LJPC-401

SAN DIEGO--(BUSINESS WIRE)--La Jolla Pharmaceutical Company (Nasdaq: 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, 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.

“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.”

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.

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 the European Medicines Agency’s Innovation Task Force

The European Medicines Agency (EMA) established its Innovation Task Force (ITF) in order to provide support for medical innovation in the European Union (EU), with a particular focus on emerging therapies and technologies. 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.

About Beta Thalassemia

Beta thalassemia is a disease characterized by a genetic mutation that results in the underproduction of hemoglobin, the body’s natural oxygen-carrying molecule contained in red blood cells. There are three types of beta thalassemia: beta thalassemia minor, beta thalassemia intermedia and beta thalassemia major. Patients with the more severe forms (intermedia and major) suffer from severe and sometimes life-threatening anemia, bone deformities and enlargement of the spleen. Standard treatment includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of 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.

About Sickle Cell Disease

Sickle cell disease is the most common inherited blood disorder in the United States. Sickle cell disease is characterized 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. Patients with severe forms suffer from severe and sometimes life-threatening anemia, strokes, and damage to vital organs such as the lungs, spleen, kidney and liver. Standard treatment includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of 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.

About Hereditary Hemochromatosis

Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body’s naturally occurring regulator of iron absorption and distribution. Without proper levels of hepcidin, excessive amounts of iron accumulate in the body and can lead to liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes. With no FDA-approved treatments for HH, patients are treated with iron chelators and phlebotomy, which do not address the underlying disease pathology, carry significant toxicity and/or adversely impact quality of life.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. Hepcidin prevents excessive iron accumulation in tissues, 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 hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. HH is a disease caused by a genetic deficiency in hepcidin that results 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. Beta thalassemia and sickle cell disease are genetic diseases of the blood that can cause life-threatening anemia and usually require frequent and life-long blood transfusions. These blood transfusions cause excessive iron accumulation in the body, which is toxic to vital organs, such as the liver and heart.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla’s Investigational New Drug Application (IND) has been accepted by the FDA, and La Jolla expects to release preliminary results from a Phase 1 study by the end of 2015.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND), commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; potential indications for which the Company’s drug candidates may be developed; and the Company’s ability to obtain the potential benefits of orphan drug designation for its drug candidates. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.