GlobalData : FDA approval for Madrigal’s Rezdiffra in metabolic dysfunction-associated steatohepatitis will set precedence for approval of future pipeline agents, says GlobalData

Following the news on 14 March 2024 that FDA has granted accelerated approval for Madrigal Pharmaceuticals' thyroid hormone receptor (THR)-β agonist Rezdiffra (resmetirom) for the treatment of patients with metabolic dysfunction-associated steatohepatitis (MASH), also known as nonalcoholic steatohepatitis (NASH), with moderate to advanced liver fibrosis;

Sravani Meka MPH, Senior Pharma Analyst at GlobalData, a leading data and analytics company, offers her view:

"In an indication where there were previously no approved therapies and multiple development failures, the approval of Rezdiffra will now set the standard for all other treatments in development. Future therapies will now have to meet or outperform efficacy and safety data generated from resmetirom's Phase III MAESTRO studies (MAESTRO-NASH (significant fibrosis), MAESTRO-NASH Outcomes (compensated cirrhosis), and MAESTRO-NAFLD-1 (safety). According to GlobalData estimates, the NASH/MASH market is anticipated to reach $25.7 billion in 2032 across the seven major markets (the US, France, Germany, Italy, Spain, the UK, and Japan). With competitor assets projected to launch during the forecast period from 2024 to 2032, including but not limited to Novo Nordisk's semaglutide, Lily's tirzepatide, and Viking's (THR)-β agonist VK-2809, Rezdiffra's approval gives Madrigal the opportunity to reap the benefits of the first-to-market therapy for patients with non-cirrhotic MASH with stages F2 to F3 fibrosis. However, it remains to be seen how long Madrigal will be able to hold this lead as we see the entry of the incretin-modulating therapies into MASH during the forecast period.

"According to Madrigal, FDA has approved the prescription of Rezdiffra without the requirement of a liver biopsy for diagnosis. This is another major win for Madrigal as the non-requirement of biopsy will aid in the uptake of this therapy. Prior to the approval, a significant point of contention in the industry revolved around the potential requirement for a biopsy, which sparked concerns that the requirement of this procedure could restrict access to Rezdiffra.

"Having overcome the barrier of FDA approval, Madrigal has been taking strategic steps to ramp up the commercialization of Rezdiffra and educating patients, healthcare professionals (HCPs), and payers about this therapy and its significance in MASH treatment. The company has already launched multiple awareness campaigns for patients and healthcare professionals, including its "#1 Liver Fan" (view here) and "NASH explored" (view here), encouraging patients to take their liver health seriously. Additionally, Madrigal has made several leadership changes and engaged in a series of financing rounds to support resmetirom through the remaining clinical stages and into the planned commercial activities.

"As Madrigal continues its journey in commercializing resmetirom, it would not be surprising if the company is also looking to position itself for an acquisition or partnership within the NASH landscape, further heightening anticipation and excitement surrounding its progress."