The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted on Friday a positive opinion recommending approval for Madrigal Pharmaceuticals, Inc.’s MDGL resmetirom (Rezdiffra) for adults with noncirrhotic MASH with moderate to advanced liver fibrosis.
EMA has recommended granting a conditional marketing authorization, said a press release.
The European Commission decision is anticipated in August.
Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH), is a serious liver disease that can progress to cirrhosis, liver failure, liver cancer, need for liver transplantation and premature mortality.
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The CHMP opinion was based on resmetirom’s favorable profile, including the positive results from the pivotal Phase 3 MAESTRO-NASH trial, which achieved both fibrosis reduction and MASH resolution primary endpoints.
The U.S. Food and Drug Administration (FDA) granted accelerated approval in March 2024 for Rezdiffra in conjunction with diet and exercise for the treatment of adults with noncirrhotic MASH with moderate to advanced liver fibrosis.
Continued approval for this indication may be contingent upon verification and description of clinical benefit in ongoing confirmatory trials.
In May, Madrigal Pharmaceuticals announced two-year results from the open-label compensated MASH cirrhosis (F4c) arm of the Phase 3 MAESTRO-NAFLD-1 trial of Rezdiffra.
Patients (n=122) in the study achieved significant improvements from baseline in liver stiffness, liver fat, fibrosis biomarkers, liver volume and risk scores for clinically significant portal hypertension (CSPH).
MAESTRO-NAFLD-1 included an open-label active treatment arm of patients with compensated MASH cirrhosis.
Among patients with CSPH at baseline, 65% moved into lower-risk categories by year two (42% to no/low CSPH and 23% to probable CSPH). Among patients with probable CSPH at baseline, 57% moved into the no/low CSPH category as compared to 14% who moved into the CSPH category by year two.
Improvement in CSPH risk was statistically significant compared to baseline.
As previously reported, patients achieved a mean 6.7 kPa reduction in liver stiffness at two years, statistically significant compared to baseline. In a responder analysis examining ≥25% improvement or worsening of liver stiffness, 51% of patients achieved improvement.
An improvement of this magnitude has been associated with reduced progression to end-stage liver disease.
Rezdiffra helped 35% of patients achieve liver stiffness measurements consistent with F3 fibrosis, suggesting reversal of cirrhosis.
The company generated first-quarter 2025 net revenues of $137.3 million. No product sales were recognized during the comparable prior year period.
MDGL Price Action: Madrigal Pharmaceuticals stock is up 0.33% at $283.66 at publication on Friday.
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