New Drug To Treat Hereditary Hemochromatosis Approved for Testing in US

Posted August 14th, 2015 by Administrator

La Jolla, a US pharmaceutical company has just received clearance from the Federal Drug Administration (FDA) to begin a Phase 1 study of LJPC-401, a novel formulation of hepcidin which is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, 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 (HHC). HHC is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HHC is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. Specifically, La Jolla has completed animal toxicology studies that demonstrated a dose-dependent reduction in serum iron levels in all species tested. La Jolla expects to release preliminary results from a Phase 1 clinical trial of LJPC-401 by the end of 2015.

It is estimated the 80,000 Canadians have Heredity Hemochromatosis but are unaware they have the condition. The Mission of the Canadian Hemochromatosis Society is to promote the early diagnosis of hereditary hemochromatosis by raising public awareness, informing the medical community, and encouraging early screening of at-risk populations. The CHS also provides support and information to individuals and families affected by the condition.