Everything to Know About Rezdiffra, the First FDA-Approved Fatty Liver Drug
Find out how the new fatty liver disease drug works, who qualifies and its potential side effects
For those dealing with fatty liver disease, there’s welcome news to report. Earlier this year, the United States Food and Drug Administration (FDA) approved the first and only fatty liver disease drug to be used by adults with non-alcoholic steatohepatitis (NASH). Keep reading to find out more about Rezdiffra (resmetirom) and whether it might be right for you.
How the FDA-approved fatty liver disease drug works
Meant to be used in conjunction with a healthy diet and exercise, Rezdiffra is designed to treat people with NASH with moderate to advanced liver scarring (fibrosis), but not with cirrhosis (severe scarring) of the liver.
NASH is a form of nonalcoholic fatty liver disease (NAFLD), which occurs when harmful, visceral fat makes up more than 5 percent of the liver’s mass, explains Supriya Rao, MD, Board-Certified Obesity Doctor, Gastroenterologist and Director of Medical Weight Loss at Lowell General Hospital and Clinical Assistant Professor at Tufts University School of Medicine.
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“Over time, NAFLD can progress to chronic inflammation and scarring. Of those with NAFLD, about 20 percent have NASH, which occurs when there is inflammation and liver cell injury,” she explains. “And 25 percent of those with NASH may have cirrhosis (irreversible scarring), which can lead to liver cancer and other organ system failure.”
How does Rezdiffra help? The drug is a thyroid hormone receptor that activates receptors in the liver to facilitate reduction in fat, inflammation and scarring, says James Burton, MD, who oversees liver transplants as a transplant hepatologist at UCHealth and faculty member at the University of Colorado School of Medicine.
“The drug is a targeted therapy that directly addresses the metabolic dysfunction in the liver in patients with metabolic dysfunction-associated steatohepatitis (MASH),” Dr. Burton explains. In trials, the fatty liver drug was found to reduce fat and inflammation and improve fibrosis.
Clinical trial results for the fatty liver disease drug
The safety and efficacy of Rezdiffra was tested in a 54-month, randomized, double-blind placebo-controlled trial, which means that participants were randomly assigned to receive either the drug or a placebo without knowing which they were receiving, to help ensure unbiased results. According to the FDA, a surrogate endpoint was used—meaning results were predicted 12 months into the trial—to measure the extent of liver inflammation and scarring.
Rezdiffra was evaluated under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and address an unmet medical need. The manufacturer of Rezdiffra is still required to conduct a post-approval study to verify the drug’s clinical benefit by completing the same 54-month study, which is currently ongoing.
To enroll in the trial, patients needed to have a liver biopsy showing inflammation due to NASH with moderate or advanced liver scarring. In the trial, 888 participants were randomly assigned to receive either a placebo, 80 milligrams of Rezdiffra or 100 milligrams of Rezdiffra once daily. That was in addition to standard care for NASH, which included counseling for a healthy diet and physical activity.
At 12 months, liver biopsies showed that a greater proportion of subjects who were treated with Rezdiffra achieved NASH resolution or an improvement in liver scarring compared to those who received a placebo. Up to 36 percent of those who received Rezdiffra experienced NASH resolution and no worsening of liver scarring, compared to just 9 to 13 percent of those who received a placebo and counseling on diet and exercise alone.
Additionally, up to 28 percent of people who received Rezdiffra experienced an improvement in liver scarring and no worsening of NASH, compared to 13 to 15 percent of those who received a placebo. In other words, the new fatty liver drug seems to work.
Rezdiffra side effects and key considerations for use
Trials found the most common side effects of Rezdiffra were diarrhea and nausea. The drug also comes with a warning of potential drug-induced liver toxicity and gallstones or gallbladder/pancreas inflammation including vomiting, fever or stomach pain.
The drug manufacturer warns that people with severe liver damage (cirrhosis) should not take Rezdiffra, and should proceed with caution taking the medication simultaneously with other drugs (specifically statins for lowering cholesterol).
Dr. Burton also notes that the FDA limited the drug to those with stages 2 and 3 fibrosis, but did not specify how to determine who has this degree of fibrosis.
“This is a potential problem, as I’ve already seen several patients prescribed the drug for second opinions who did not meet indications for having high-risk MASH or evidence of moderate to severe fibrosis on liver biopsy,” he explains. “It is my current practice to recommend a biopsy in anyone with suspected MASH who might be suspected of having advanced fibrosis on non-invasive testing to at least ensure an accurate baseline.”
Is the fatty liver disease drug right for you?
Rezdiffra can be helpful for those who meet the qualifications. Some insurance plans will partially cover the fatty liver disease drug, but there may still be significant out-of-pocket costs—and the drug costs an astonishing $46,000 per year, according to a press release about the Rezdiffra. To help offset the high price tag, Rezdiffra offers a Copay Savings Card and those who are eligible may be able to get the fatty liver disease drug for as little as $10 a month.
It’s also important to remember that diet and exercise play a key role in preventing and reversing fatty liver disease even for those taking the drug. Rezdiffra is not a replacement for a healthy lifestyle, especially when it comes to weight loss and control of type 2 diabetes, emphasizes Dr. Burton.
The bottom line on the new FDA fatty liver disease drug
Overall, Rezdiffra is an effective treatment for fatty liver and scarring, and the drug is generally well-tolerated. If you’ve been diagnosed with fatty liver disease with moderate to advanced scarring without cirrhosis, you might want to speak with your doctor about whether Rezdiffra could be a helpful addition to a healthy lifestyle.
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