Hemochromatosis Therapeutics Pipeline to Witness Significant Growth due to Positive Clinical Results in the Coming Years | Top Key Players are Novartis, ApoPharma Inc., La Jolla Pharmaceutical Company, DisperSol Technologies, LLC, Silence Therapeutics plc

Hemochromatosis, also known as iron overload, is identified as increased level of iron in the body. It has symptoms such as joint pain, weight loss, erectile dysfunction in men, and irregular menstrual cycle in women. Hemochromatosis can be categorized into two types: primary hemochromatosis and secondary hemochromatosis. Primary hemochromatosis is genetic disorder; however, secondary hemochromatosis occurs due to medical conditions such as anemia, thalassemia, and others.

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If hemochromatosis is not diagnosed or treated at an early stage, it may lead to some serious complications such as liver damage, diabetes, arthritis, reproductive complications, and cardiovascular diseases. The commonly used diagnostic techniques are blood test, and physical examination. Additional liver function test (LFT), magnetic resonance imaging (MRI), and liver biopsy procedures are performed to confirm the disease.
Exjade by Novartis International AG, and Ferriprox by ApoPharma Inc. are the two key U.S. Food and Drug Administration (USFDA) approved drugs for the treatment of hemochromatosis.

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According to the research findings, majority of pipeline drug candidates are being developed for oral administration. It has been observed that oral route of medications is convenient, available in delayed or rapid release formulation, less risk of systemic infections, and inexpensive in nature, and also provides improved patient’s compliance. Administration of therapeutics for hemochromatosis through the oral route has shown promising results in clinical studies.

Companies that are involved in developing therapeutics for hemochromatosis have shown positive clinical results in various phases of drug development. For instance, in September 2016, La Jolla Pharmaceutical Company demonstrated positive results from a phase I study of LJPC-401 in patients with hereditary hemochromatosis, thalassemia, and sickle cell diseases. Results of the study were positive as LJPC-401 was well-tolerated with no dose limiting toxicities. Furthermore, in March 2019, La Jolla Pharmaceutical Company announced in its financial report that topline results of LJPC-401 in patients with hereditary hemochromatosis are expected in second half of 2019.

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As of 2018, Exjade and Ferriprox are two marketed products available in the market for the treatment of hemochromatosis. Due to emergence of late- and mid-stage pipeline products, the overall hemochromatosis therapeutics market is expected to grow significantly in the upcoming years.
Novartis International AG, ApoPharma Inc., La Jolla Pharmaceutical Company, DisperSol Technologies, Silence Therapeutics plc, and Vifor Pharma Management Ltd. are some of the key companies involved in the development of drug candidates for the treatment of hemochromatosis.

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