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news.The US Food and Drug Administration (FDA) has accepted La Jolla Pharmaceutical's investigational new drug application (IND) for its new formulation of hepcidin, LJPC-401, being developed for the treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia.
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LJPC-401 is being evaluated in a Phase I clinical trial and the company intends to release preliminary results from this trial by the end of this year.
Hepcidin regulates the absorption and distribution of iron to prevent excessive iron accumulation in tissues, such as the liver and heart, where it can cause significant damage and even result in death.
HH is a disease caused by a genetic deficiency in hepcidin and is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.
La Jolla president and chief executive officer George Tidmarsh said: "We are pleased to have received clearance from the FDA to begin a Phase I study of LJPC-401.
"LJPC-401 presents a unique opportunity to potentially help patients suffering from the effects of iron overload by restoring normal or near-normal levels of hepcidin, the body’s natural regulator of iron absorption and distribution."
The company noted that in preclinical testing, LJPC-401 has been shown to be effective in reducing serum iron.
Specifically, the company has completed animal toxicology studies that showed a dose-dependent reduction in serum iron levels in all species tested.