FDA’s Approval of Breakthrough NASH Treatment Opens Door for a Long-Neglected Patient Population
GLI Celebrates Approval of Madrigal Pharmaceutical’s Rezdiffra™ (resmetirom) as Milestone for Liver Health
(Washington D.C., March 14, 2024) – Today, Global Liver Institute (GLI) recognizes the importance of the first approval by the U.S. Food and Drug Administration (FDA) of a medication for nonalcoholic steatosis (NASH). Developed by Madrigal Pharmaceuticals, Rezdiffra™ (resmetirom) was approved for the treatment of adults with noncirrhotic NASH with moderate to advanced liver fibrosis, in conjunction with diet and exercise. This breakthrough follows years of NASH patient-led, multi-stakeholder advocacy and partnership with researchers in both drug and diagnostic development. The hope of the NASH and larger liver health community is that this successful approval and the robust and diverse pipeline of therapies to follow will open up a new era of care options for people living with fatty liver disease.
“Today’s announcement represents the culmination of the work of the united NASH community. It is particularly satisfying that we can mark Women’s History Month with a breakthrough from a female-founded company. We feel validated in establishing the GLI NASH Council back in 2017 as a collaboration that included endocrine and cardiology groups, organizations that served minority communities, and others outside hepatology from the beginning, anticipating that tackling fatty liver disease would need a larger and more diverse cadre of supporters than any other liver condition had had before,” noted Donna R. Cryer, JD, founder and CEO of GLI. “While today marks a significant step, this is only the beginning of our work to meet the needs of the full spectrum of patients with fatty liver disease, which range from clarification of the language used, to simplification of the methods to screen and risk-stratify patients, and additional medications given the diversity and complexity of fatty liver disease.”
Rezdiffra™ has been approved in the United States for adults with NASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis) in conjunction with diet and exercise. As noted by the U.S. FDA, approximately 6-8 million people in the U.S. currently have NASH with moderate to advanced liver scarring.
“Treating NASH is so important because it is the fastest-growing cause of cirrhosis, liver cancer, and liver transplant. With researchers anticipating 27 million cases of NASH in the U.S. by 2030, we are thrilled with this FDA approval of the first-ever medication for NASH as another tool to treat a stage of this serious, progressive disease,” shared Jeff McIntyre, VP of Liver Health Programs at GLI. “NASH is most common among people with obesity and/or type 2 diabetes. As these conditions have become more prevalent, the risk for NASH also increased. The FDA not requiring a liver biopsy when prescribing this medication reflects the Agency’s responsiveness to the voices of patients from GLI’s Externally-Led, Patient-Focused Drug Development Meeting in 2022 and the impact of our Beyond the Biopsy™ collaborative. This approval gives patients and health care providers a long-awaited tool to change the trajectory of their chronic liver disease.”
While today’s announcement is rightfully exciting, patient advocates need to focus on major next steps. First, patients need access. The medication must be broadly available and affordable for patients across different geographies and healthcare settings. Around the globe, patients now turn their attention to the consideration of approval, starting with the EMA’s evaluation.
Once the medication is available, patients, particularly those already diagnosed with noncirrhotic NASH with moderate to advanced liver fibrosis and those with risk factors such as type 2 diabetes, obesity, high cholesterol, and/or hypertension, should ask their healthcare providers if this medication is right for them. GLI is committed to supporting all patients to learn more about their liver health, understand if they are within the eligible population for this treatment, and connect to clinical trials, peer support and other resources, most available in multiple languages.
About Global Liver Institute
Global Liver Institute (GLI) is a 501(c)3 nonprofit organization founded in the belief that liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver illness. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to help eradicate liver diseases. Operating globally, GLI is committed to solving the problems that matter to liver patients and equipping advocates to improve the lives of individuals and families impacted by liver disease. GLI holds Platinum Transparency with Candid/GuideStar, is a member of the National Health Council, and serves as a Healthy People 2030 Champion. Follow GLI on Facebook, Instagram, LinkedIn, and YouTube or visit www.globalliver.org. GLI is the host of Global Fatty Liver Day.
