Hemochromatosis/Iron Overload Therapeutics Pipeline to Witness Significant Growth | Top Players are: Novartis, ApoPharma Inc., La Jolla Pharmaceutical Company, DisperSol Technologies, LLC, Silence Therapeutics plc, Vifor Pharma

Even though, iron plays the important role of transporting oxygen in the body, its excess leads to iron overload, which can be damaging for organs, such as the heart, liver, and pancreas as well as joints and endocrine glands. The condition is called hemochromatosis, which is of three types — primary, secondary, and neonatal. Having a genetic origin, primary hemochromatosis is caused by an abnormal human hemochromatosis protein (HFE) gene. The function of this gene is to absorb the iron present in the food. The gene is rendered abnormal due to the H63D or C282Y mutation.

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On the contrary, secondary hemochromatosis isn’t inherited and is caused by external factors such as anemia and multiple blood transfusions in people suffering from severe thalassemia. Neonatal hemochromatosis is a rare disease that causes liver failure and death in fetuses and newborns.

People with mild iron overload may or may not exhibit any symptom. For severe iron overload, symptoms are high sugar level, joint pain, fatigue, weight loss, bronze coloration of the skin, and menopause in women. Complications include hepatocellular carcinoma, liver cirrhosis, cardiomyopathy, and diabetes. Hemochromatosis is diagnosed via genetic testing, which helps know the presence of the mutated HFE gene. Blood tests, such as the serum ferritin and transferrin saturation tests, may be conducted to diagnose the condition by determining the amount of iron stored in the body.

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Hemochromatosis is treated by the removal of excess iron in the body. This is done by using a technique called phlebotomy, which a safe and inexpensive way of managing the condition. At regular intervals, the blood is removed from the body that leads to a decrease in the iron level. Another mode of treatment is the chelation therapy, in which certain drugs are used that bind to the iron in the blood and remove it from the body. Deferoxamine, Deferasirox, and Deferiprone are the drugs used in iron chelation therapy.

Even though the treatment of hemochromatosis is available, these are the only three iron chelators currently available in the market. The limited availability of drugs in the market is motivating pharmaceutical companies to invest heavily in the hemochromatosis/iron overload therapeutics pipeline and take advantage of the unexplored opportunities. This is acting as a key driving factor for the market across the world.

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Another major factor contributing to the growth of the hemochromatosis/iron overload therapeutics pipeline is the side effects associated with the drugs. The chelators may cause gastrointestinal hemorrhage, kidney failure, and liver failure. For instance, Deferasirox treatment may lead to proximal renal tubular dysfunction. In older patients, this drug may lead to the predisposition of renal or hepatic disease or myelodysplastic syndrome. Further, Deferoxamine infusions in children are painful and time-consuming, which is further helping the pipeline grow in a bid to find more effective and safer treatment alternatives.

One of the drug candidates in the hemochromatosis/iron overload therapeutics pipeline is LJPC-401, an under-investigation clinical-stage product. A trademark formulation being tested by PRA Health Sciences Inc. and La Jolla Pharmaceutical, it is a synthetic human hepcidin. Hepcidin is an endogenous peptide hormone, which is the body’s natural regulator of iron absorption and distribution. In healthy individuals, it prevents excessive iron accumulation in organs such as the heart and liver.

The company is pursuing this candidate as a potential treatment alternative for primary as well as secondary hemochromatosis. LJPC-401 has been designated as an orphan medicinal product by the European Medicines Agency Committee for Orphan Medicinal Products for the treatment of sickle cell disease, and beta thalassemia intermedia and major. An orphan drug is one which is produced with government assistance, because the disease it treats is so rear that pharma companies are reluctant to develop it.

Therefore, it is clear that government assistance is essential for the growth of the therapeutics pipe line for hemochromatosis.

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This release was published on openPR.