Credit: Getty Images/ Hiroshi Watanabe

The FDA has granted Madrigal Pharmaceuticals’ resmetirom a Breakthrough Therapy designation for nonalcoholic steatohepatitis (NASH) with liver fibrosis, as the company completes enrolment of the outcomes portion of a Phase III trial.

Following the company’s announcement on April 18, Madrigal’s stock volume more than doubled itself compared to the previous day. Furthermore, the company’s stock price closed at 12% higher than at the start of the day.

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In January 2023, Madrigal received attention surrounding resmetirom when it released Phase III data. This was shortly followed by a February 2023 draft assessment by the Institute for Clinical and Economic Review (ICER) that found that resmetirom could be a more cost-effective NASH treatment than Intercept Pharmaceuticals’ obeticholic acid. The two companies are competing to become the first marketed NASH treatment in the US. Intercept’s obeticholic acid is a FXR agonist that treats patients with pre-cirrhotic liver fibrosis due to NASH. Obeticholic acid is currently sold at lower dosages under the brand name Ocaliva for primary biliary cholangitis.

In 2020, the FDA rejected Intercept’s application for Ocaliva’s use in NASH, causing the company to revise its Phase III protocol. The agency has since accepted Intercept’s resubmitted NDA on 19 January 2023 and scheduled a PDUFA date for 22 June 2023. Since then, the FDA has announced that it will hold an it will convene a Gastrointestinal Drugs Advisory Committee to evaluate the drug’s prospects.

Madrigal’s CEO Dr. Paul Friedman said, “The Breakthrough Therapy designation for resmetirom reflects the FDA’s recognition of resmetirom’s potential in the treatment of NASH as a serious, life-threatening condition and an important area of investigational drug development”. A Breakthrough Therapy designation expedites the drug development and review process for serious or life-threatening conditions. Resmetirom is a once-daily oral thyroid hormone receptor-beta agonist that works by lowering the effects of thyroid hormones. The CEO said Madrigal expects to approach a new drug application (NDA) filing for resmetirom in Q2 2023. This filing will be supported by positive results from the MAESTRO-NASH trial.

Madrigal released topline results from the MAESTRO-NASH trial in December 2022. The 52-week study found that patients showed statistically significant improvement in the study’s primary endpoints after taking the drug. These endpoints included measures of NASH resolution and improvement of fibrosis stage. Furthermore, the drug was well tolerated by patients.

The company also announced that it has now completed enrollment of the outcomes portion of its Phase III MAESTRO-NASH trial. Madrigal is currently running four Phase III trials to investigate the safety and efficacy of resmetirom for the treatment of NASH, MAESTRO-NASH, MAESTRO-NAFLD-1 (NCT04197479), MAESTRO-NAFLD-OLE (NCT04951219), and MAESTRO-NASH-OUTCOMES (NCT05500222). Data from these studies, along with Phase I and II studies, will form the basis of a planned FDA submission for accelerated approval of resmetirom for treatment of NASH.

Madrigal’s CMO Dr. Becky Taub said, “Enrollment of the MAESTRO-NASH study was closed at approximately 1,750 patients based on the enrollment target of the 54-month long-term clinical outcome portion of the study. Patient recruitment remains focused on MAESTRO-NASH-OUTCOMES, a Phase 3 clinical outcome trial evaluating resmetirom in patients with well-compensated NASH cirrhosis that provides a second and potentially earlier opportunity to support full approval for both noncirrhotic and cirrhotic NASH.”