Source – Caribbean News Global
In a significant development, the US Food and Drug Administration (FDA) announced on Thursday the approval of the first medication for nonalcoholic steatohepatitis (NASH treatment), a prevalent form of liver inflammation. Known medically as metabolic dysfunction-associated steatohepatitis (MASH), this condition arises from the inflammation of the liver due to an excess accumulation of fat cells.
NASH treatment represents an advanced stage of nonalcoholic fatty liver disease and affects an estimated 6 to 8 million individuals in the United States, particularly those with moderate to advanced liver fibrosis. Complications associated with NASH include cirrhosis, liver failure, and liver cancer, underscoring the urgent need for effective treatment options.
Resmetirom: A Breakthrough in NASH treatment
Until now, treatment strategies for NASH predominantly centered on lifestyle modifications, including weight loss through measures like blood sugar control, a balanced diet, and regular exercise. However, the approval of Madrigal Pharmaceuticals’ resmetirom, to be marketed as Rezdiffra, signals a paradigm shift in NASH therapy. This medication, which received Breakthrough Therapy designation from the FDA in April, operates by activating a thyroid hormone receptor in the liver, thereby aiding in the reduction of fat accumulation. Administered orally on a daily basis, Rezdiffra is specifically indicated for individuals with NASH and moderate to advanced fibrosis, complementing the importance of maintaining a healthy lifestyle.
Clinical Efficacy and Post-Approval Plans
Clinical trials of resmetirom have yielded promising results, with participants experiencing significant improvements. Notably, over 25% of those receiving 80 milligrams of the medication achieved NASH resolution without worsening fibrosis, while nearly 30% of those on 100 milligrams saw similar outcomes. Additionally, Rezdiffra demonstrated efficacy in lowering LDL cholesterol levels and liver enzyme levels.
Despite mild to moderate adverse events such as diarrhea and nausea reported during trials, the FDA granted accelerated approval for the drug, contingent upon completion of a post-approval study to confirm its clinical benefits. Wayne Eskridge, CEO of the Fatty Liver Foundation, expressed optimism about the approval, anticipating enhanced efforts in disease education, care pathways, and research investment within the NASH community. Madrigal Pharmaceuticals has announced plans for Rezdiffra’s availability next month, accompanied by an assistance program aimed at facilitating access for uninsured individuals.
The approval of Rezdiffra marks a significant advancement in NASH treatment, offering hope to millions affected by this debilitating condition and paving the way for future innovations in liver health management.
