Non-Alcoholic Steatohepatitis (NASH) Updates

Non-Alcoholic Steatohepatitis (NASH) is characterized by fat accumulation in the liver that causes inflammation and cell damage without regard to alcohol use. If you would like to know more about NASH, please click here to read our previous blog post.

The FDA has not approved any medications to treat NASH. This condition is important to identify early on and treat by means of lifestyle modifications. Due to the prevalence of NASH and the possibility that an afflicted patient could progress to cirrhosis or even liver cancer, drug manufacturers have been studying products that could be of potential use. There are a few pharmacological treatments close to entering the market – maybe as early as this year.

For patients unable to lose enough weight or maintain weight loss, pharmacological treatment is necessary. This is particularly true if a patient has progressed to the later stages of NASH, as they are at a higher risk for serious complications (i.e. cirrhosis, liver failure, and, in some cases, liver cancer). Some patients have used pioglitazone or vitamin E off label, but there is not enough data to support recommending these products. Benefits and risks must be weighed for individual patients.

Genfit and Intercept have manufactured the two most promising treatment options. Genfit is in Phase 3 studies for its drug elafibranor, a peroxisome proliferator-activated receptor (PPAR) alpha/delta dual agonist. PPARs are nuclear receptors that play major roles in the development of NASH and its comorbidities. Pioglitazone is a PPAR-gamma agonist. Activation of the gamma receptor is associated with weight gain, edema, and fluid retention, making it a less viable option.

Intercept is in Phase 3 studies for its drug obeticholic acid (OCA). The company filed an NDA for FDA approval in September of 2019. OCA is a farnesoid X-receptor (FXR) agonist. OCA is available as brand name product, Ocaliva, indicated for use in the treatment of biliary cholangitis. A month of treatment is estimated to cost $7,000.00, making it an expensive option. Although this drug has displayed the ability to stabilize and sometimes reverse the features of NASH, its high cost will be a clear limitation to its widespread use.

Due to the lack of successful treatment options, drug manufacturers are anxious to launch approved products to serve the NASH market of over 300 million potential patients worldwide. The question is, when a drug does hit the market, will payers cover it, and will patients be able to afford it? The first step to accessing treatment will likely be a diagnosis of NASH and fibrosis staging through a liver biopsy. Payers will need to weigh the cost of these new medications against the cost of alternatives, such as bariatric surgery or a liver transplant. There are few existing alternatives, and if a patient has damage that is too severe, they will likely require medication.

At this time, healthcare professionals should recommend lifestyle modifications and emphasize the need to control comorbid conditions, such as diabetes, hypertension and dyslipidemia. FDA-approved drugs will likely be available in the near future – stay up to date and knowledgeable!

 

Posted April 2020

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