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 5. FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty
    Liver Disease

 1. Press Announcements

FDA News Release


FDA APPROVES FIRST TREATMENT FOR PATIENTS WITH LIVER SCARRING DUE TO FATTY LIVER
DISEASE

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For Immediate Release: March 14, 2024



Español

Today, the U.S. Food and Drug Administration approved Rezdiffra (resmetirom) for
the treatment of adults with noncirrhotic non-alcoholic steatohepatitis (NASH)
with moderate to advanced liver scarring (fibrosis), to be used along with diet
and exercise. 

“Previously, patients with NASH who also have notable liver scarring did not
have a medication that could directly address their liver damage,” said Nikolay
Nikolov, M.D., acting director of the Office of Immunology and Inflammation in
the FDA’s Center for Drug Evaluation and Research. “Today’s approval of
Rezdiffra will, for the first time, provide a treatment option for these
patients, in addition to diet and exercise.” 

NASH is a result of the progression of nonalcoholic fatty liver disease where
liver inflammation, over time, can lead to liver scarring and liver dysfunction.
NASH is often associated with other health problems such as high blood pressure
and type 2 diabetes. By at least one estimate, approximately 6-8 million people
in the U.S. have NASH with moderate to advanced liver scarring, with that number
expected to increase. Rezdiffra is a partial activator of a thyroid hormone
receptor; activation of this receptor by Rezdiffra in the liver reduces liver
fat accumulation. 

The safety and efficacy of Rezdiffra was evaluated based on an analysis of a
surrogate endpoint at month 12 in a 54-month, randomized, double-blind
placebo-controlled trial. The surrogate endpoint measured the extent of liver
inflammation and scarring. The sponsor is required to conduct a postapproval
study to verify and describe Rezdiffra’s clinical benefit, which will be done
through completing the same 54-month study, which is still 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 subjects were
randomly assigned to receive one of the following: placebo (294 subjects); 80
milligrams of Rezdiffra (298 subjects); or 100 milligrams of Rezdiffra (296
subjects); once daily, in addition to standard care for NASH, which includes
counseling for healthy diet and exercise. 

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 as compared with those who received the placebo. A total of 26% to 27%
of subjects who received 80 milligrams of Rezdiffra and 24% to 36% of subjects
who received 100 milligrams of Rezdiffra experienced NASH resolution and no
worsening of liver scarring, compared to 9% to 13% of those who received placebo
and counseling on diet and exercise. The range of responses reflects different
pathologists’ readings. In addition, a total of 23% of subjects who received 80
milligrams of Rezdiffra and 24% to 28% of subjects who received 100 milligrams
of Rezdiffra experienced an improvement in liver scarring and no worsening of
NASH, compared to 13% to 15% of those who received placebo, depending on each
pathologist’s readings. Demonstration of these changes in a proportion of
patients after just one year of treatment is notable, as the disease typically
progresses slowly with a majority of patients taking years or even decades to
show progression.

The most common side effects of Rezdiffra included diarrhea and nausea.
Rezdiffra comes with certain warnings and precautions, such as drug-induced
liver toxicity and gallbladder-related side effects. 

Use of Rezdiffra should be avoided in patients with decompensated cirrhosis.
Patients should stop using Rezdiffra if they develop signs or symptoms of
worsening liver function while on Rezdiffra treatment. 

Using Rezdiffra at the same time as certain other drugs, in particular statins
for lowering cholesterol, may result in potentially significant drug
interactions. Health care providers should refer to the full prescribing
information  for additional information on these potentially significant drug
interactions with Rezdiffra, recommended dosage and administration
modifications. 

The FDA approved Rezdiffra under the accelerated approval pathway, which allows
for earlier approval of drugs that treat serious conditions and address an unmet
medical need, based on a surrogate or intermediate clinical endpoint that is
reasonably likely to predict clinical benefit. The required aforementioned
54-month study, which is ongoing, will assess clinical benefit after 54 months
of Rezdiffra treatment. 

Rezdiffra received Breakthrough Therapy, Fast Track and Priority Review
designations for this indication. 

The FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.


RELATED INFORMATION

Related Information
 * NIH: Nonalcoholic Fatty Liver Disease (NAFLD) & NASH

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