Madrigal’s NASH drug gets fast-track review from FDA, no panel needed

Madrigal Pharmaceuticals, NASH, resmetirom, FDA, priority review, nonalcoholic steatohepatitis

In the wake of a recent CEO transition, Madrigal Pharmaceuticals has disclosed that its nonalcoholic steatohepatitis (NASH) drug has been granted priority review by the FDA, and the agency does not intend to convene an advisory committee meeting.

Madrigal had submitted a new drug application seeking accelerated approval for resmetirom, an oral medication designed for daily use in patients with liver fibrosis and NASH, the most severe manifestation of nonalcoholic fatty liver disease. The FDA has set a decision date for March 14, 2024, and has dispensed with the need for an advisory committee meeting.

If granted approval, resmetirom could make history as the first treatment for this condition. Remarkably, only four investigational treatments have progressed to phase 3 development for NASH, and resmetirom is leading the pack.

Notably, the FDA’s decision to forgo an advisory committee meeting is raising eyebrows. This move is considered somewhat unusual, particularly for a disease like NASH, which still lacks an approved drug. Analysts view this development as “an incremental derisking event suggesting that FDA has all the elements it needs to complete the review,” according to Evercore analyst Liisa Bayko.

This news follows closely on the heels of Madrigal’s former CEO, Paul Friedman, M.D., stepping down from his position. Bill Sibold has assumed the role as the company prepares to transition into a commercial entity. Sibold brings with him a wealth of experience, having spent 12 years at Sanofi, most recently serving as Executive Vice President of Specialty Care and President of North American Operations.

“NASH with liver fibrosis represents a significant unmet need in healthcare today: the disease has a serious impact on patients and, without treatment, it can lead to increased risk of cirrhosis, liver failure, liver cancer, and premature mortality. Resmetirom is a liver-directed therapy that has demonstrated the potential to treat the liver fibrosis that is associated with these negative outcomes, while resolving the underlying steatohepatitis that drives the disease. The FDA’s acceptance of our NDA with priority review is an important step forward as we pursue our goal of delivering the first approved treatment to patients with NASH with liver fibrosis.”

– Bill Sibold, Chief Executive Officer of Madrigal

In response to these developments, Madrigal’s stock, which experienced a slight dip following the CEO change, has rebounded. As of 10:30 a.m. ET on Wednesday, it has risen from $177 at market close the previous day to $189 per share.

Madrigal Pharmaceuticals is now standing at the cusp of a potentially groundbreaking moment in NASH treatment, and the FDA’s prioritized review signals a significant step forward. The leadership transition at the company further underscores the pivotal juncture it finds itself in as it prepares for potential commercialization and the transformative impact it could have on patients battling this challenging condition.

“We believe that we have delivered a compelling data package to support the FDA’s benefit-risk evaluation of resmetirom for the treatment of NASH with liver fibrosis. The NDA is supported by the positive efficacy results observed in our pivotal Phase 3 trial, the large safety database we have established through the MAESTRO program, and two ongoing outcomes studies that are designed to verify clinical benefit following a potential accelerated approval. We look forward to beginning this critical next phase of the review process.”

– Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal

Share This News