HealthBreakthrough: FDA Greenlights First Drug for Severe Liver Condition

Breakthrough: FDA Greenlights First Drug for Severe Liver Condition

Treatment for Liver Scarring in NASH Patients

Written by: Jenna Smith, Health Correspondent

The recent approval by the FDA of resmetirom (Rezdiffra) marks a significant milestone in the treatment of noncirrhotic non-alcoholic steatohepatitis (NASH), now referred to as metabolic dysfunction-associated steatohepatitis (MASH). This once-daily oral medication targets key underlying causes of NASH and is specifically designed for patients with moderate to advanced liver fibrosis, ranging from stages F2 to F3 fibrosis. It is intended to be used in conjunction with a balanced diet and regular exercise.

According to a statement by Nikolay Nikolov, MD, from the FDA’s Center for Drug Evaluation and Research, patients with significant liver scarring due to NASH previously had limited treatment options. The approval of Rezdiffra now provides these patients with a direct medication to address their liver damage, complementing lifestyle changes.

NASH, a consequence of non-alcoholic fatty liver disease (NAFLD) progression, can lead to liver fibrosis, dysfunction, and increase the risk of liver transplant and hepatocellular carcinoma. It is frequently associated with comorbid conditions such as hypertension and diabetes. Current estimates suggest that over 6 million Americans may have NASH with stages F2 to F3 fibrosis, a number expected to rise in the future.

The approval of resmetirom is supported by data from the phase III MAESTRO-NASH study, which showed promising results. Patients receiving an 80mg or 100mg dose of the medication demonstrated NASH resolution without worsening of fibrosis in significant numbers compared to those on placebo. Similarly, fibrosis improvement by at least one stage with no worsening of NAFLD activity score was notable in patients receiving resmetirom.

While the MAESTRO-NASH study required patients with confirmed NASH inflammation and liver scarring to participate, the prescribing information for resmetirom does not mandate a liver biopsy for diagnosis. This flexibility may improve access to treatment for a broader patient population.

Dr. Stephen Harrison, a lead investigator in the MAESTRO study, emphasized the groundbreaking impact of this medication on healthcare providers, the research community, and patients living with NASH. He envisions resmetirom becoming a central therapy for NASH patients with moderate to advanced liver fibrosis, given its efficacy and safety profile in clinical trials.

As part of the accelerated approval process, Madrigal Pharmaceuticals will conduct a postmarketing study to further evaluate the clinical benefits of resmetirom in this patient population. This research aims to assess whether the observed positive outcomes in the MAESTRO studies translate to a reduced risk of disease progression.

In conclusion, the approval of resmetirom signifies a significant advancement in the treatment landscape for NASH patients with liver scarring. By targeting the underlying causes of the disease and showing promising clinical outcomes, this medication offers hope for improved management of this serious liver condition in the years to come.

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