First Drug to Treat Common, Lethal Liver Disease Gets US Nod

  • 16 Mar 2024

Why is it in the News?

Madrigal Pharmaceuticals Inc.’s drug Rezdiffra gained the first US approval to treat a potentially deadly liver disease that affects millions worldwide, succeeding in an area where some bigger rivals have failed.

What is Nonalcoholic Steatohepatitis (NASH)?

  • Nonalcoholic fatty liver disease (NAFLD) is a condition in which fat builds up in the liver.
  • NASH (or nonalcoholic steatohepatitis) is a type of NAFLD that can damage the liver.
  • NASH occurs when the fat buildup in the liver leads to inflammation (hepatitis) and scarring.
  • NASH can be life-threatening, as it can cause liver scarring (called cirrhosis) or liver cancer.
  • It is estimated that 3% to 5% of the global population is affected by NASH, though the disease is considered to be underdiagnosed.

Who gets NASH?

  • The condition may be hereditary.
  • If a person has family members who have had NASH or NAFLD, they are at risk.
  • Additionally, having certain health conditions may increase a person’s risk of developing NASH. These include:
    • Being overweight or obese.
    • Having high cholesterol or high triglyceride levels.
    • Having type 2 diabetes, insulin resistance, or prediabetes.

What are the signs and symptoms of NASH?

  • NASH is known as a “silent” disease because many people present with few or no symptoms.
  • However, some people will experience tiredness, pain, and discomfort in the upper right part of the abdomen.

How is NASH diagnosed?

  • Diagnosing NASH can be challenging because symptoms may not be noticeable until the disease progresses.
  • Healthcare providers typically suspect NASH based on abnormal blood or liver test results or imaging showing liver fat.
  • Confirmation requires a liver biopsy, an invasive procedure with risks and expenses, involving taking a small liver sample for microscopic examination.

How is NASH treated?

  • To manage NASH, losing weight is often recommended as it can reduce liver fat, inflammation, and scarring.
  • This involves losing around 3% to 5% of body weight by limiting fats and sugars in the diet.
  • Heavy alcohol use should also be avoided to prevent further liver damage.
  • If NASH progresses to cirrhosis, treatment may involve medications, medical procedures, or even a liver transplant.
  • Currently, there are no approved medications specifically for treating NASH, but ongoing research aims to develop new treatments.