FDA approves first drug for common form of liver inflammation

The FDA has approved the first-ever medication for nonalcoholic steatohepatitis (NASH), a common form of liver inflammation linked to obesity, type 2 diabetes, and other metabolic conditions. NASH, characterized by liver inflammation due to excess fat accumulation, affects an estimated 6 to 8 million people in the US with moderate to advanced fibrosis.

Until now, NASH treatment focused on lifestyle changes like diet and exercise. The newly approved medication, Madrigal Pharmaceuticals’ resmetirom, marketed as Rezdiffra, received Breakthrough Therapy designation for its promising potential. Administered orally daily, it activates a thyroid hormone receptor in the liver to reduce fat accumulation, recommended alongside diet and exercise for those with moderate to advanced fibrosis.

Clinical trials showed significant NASH resolution rates with resmetirom, notably reducing LDL cholesterol and liver enzyme levels. Despite mild adverse events such as diarrhea and nausea, caution is warranted due to potential interactions with statins. Rezdiffra's accelerated approval mandates further post-approval studies to confirm clinical benefits.

Wayne Eskridge, CEO of the Fatty Liver Foundation, anticipates Rezdiffra's availability next month, providing assistance programs for uninsured individuals. Dr. Pierre Gholam, a hepatologist, views the approval as a monumental advancement after decades of unsuccessful drug development for NASH, signaling hope for improved patient outcomes and future treatments.