Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Development Studies Are Increasingly Being Conducted All Over The World

Progressive Familial Intrahepatic Cholestasis Type 2 Treatment
Progressive Familial Intrahepatic Cholestasis Type 2 Treatment

PFIC stands for progressive familial intrahepatic cholestasis. This condition is autosomal recessive, which means that the affected person is healthy because they have a working copy of the gene. However, the carriers of this disease can pass on the non-working copy to their children. The symptoms of PFIC may appear in one or more of their children. Progressive Familial Intrahepatic Cholestasis Type 2 Treatment occurs in two types, type 1 and type 2. In PFCI type 2 the affected liver has multiple bile acids. It is usually caused by mutations of flippase genes, which flip phospholipids in the plasma membrane. The FXR protein is responsible for the production of bile salts, which is essential for proper digestion. Several other genes are implicated in PFIC.

A new drug for the Progressive Familial Intrahepatic Cholestasis Type 2 Treatment is being developed. In Europe, there are a few approved  PFIC Type 2 treatment options available for this condition. A number of pharmaceutical companies are focusing on research and development for novel drugs. A recently approved drug, a combination of cholestasis medications, is being tested by physicians. Several other companies are also in the process of developing new drugs for the disease.

A number of companies are developing novel Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market. Among these, Mirum Pharmaceuticals and Genevaxax are two companies focused on developing innovative therapies for these diseases. Moreover, their phase 2 indigo study, which was presented in the International Liver Congress 2020, showed a marked improvement in the patients' transplant-free survival. There are no other treatments for PFIC type 2, however, many research programs are still underway.

People with PFIC2 have a high risk of developing hepatic cirrhosis. PFIC2 patients are at a high risk of hepatocellular carcinoma and need a liver transplant. Currently, there is no treatment for PFIC2, but there are various medications available for the condition. In July 2021, The U.S. Food and Drug Administration (FDA) granted approval to Odevixibat, the first drug treatment for Progressive Familial Intrahepatic Cholestasis.

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