Updates in Experimental Therapies for Liver Disease

According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases still lack disease modifying therapies. Here is a brief list of updates from the last several months related to the development of liver disease therapies that, if successful, will hopefully give patients with these diseases greater opportunities for effective treatment and improved quality of life and overall outcomes.

Non-Alcoholic Fatty Liver Disease (NAFLD)

The drug developer Lipocine recently released a report detailing the results of a clinical trial testing the company’s investigational product candidate LPCN 1144 as a treatment for non-alcoholic fatty liver disease. This disease is not considered rare but current treatments for it are limited and it is increasing in prevalence. After a 16 week period of treatment, LPCN 1144 was able to successfully reduce liver fat. Learn more about this study here.

Acute Hepatic Porphyria

The company Alnylam Pharmaceuticals recently announced results from a Phase 3 clinical trial testing the company’s experimental candidate givosiran. This drug is being developed as a therapy for acute hepatic porphyria. In this trial, givosiran was able to successfully bring down the number of porphyria attacks when compared against a placebo. These attacks can be debilitating and life threatening, but these results suggest that givosiran could help improve the lives of patients. Read more here.

Autoimmune Hepatitis

Stero Biotechs recently announced that is will start enrolling patients with autoimmune hepatitis in a clinical trial that will test the use of cannabidiol (CBD). The goal is to use CBD in order to reduce the dosage and duration of steroid treatment in autoimmune hepatitis. Long term steroid use can have serious side effects. The trial will include 15 patients. Click here to learn more.


Imbrium Therapeutics and Mundipharma EDO released an announcement indicating that the FDA has granted the companies’ drug etoposide toniribate Orphan Drug designation as a treatment for cholangiocarcinoma, also known as biliary tract cancer. These companies plan to develop a global Phase 3 trial to continue testing this promising therapy for a rare and deadly form of cancer. Read more about it here.

These are just some of the latest developments in the field of rare liver diseases. This field of developing rapidly and hopefully will results in improved outcomes for patients in the near future.

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