FDA Approves First Medication for Nonalcoholic Steatohepatitis (NASH)
The US Food and Drug Administration (FDA) has granted its first approval for a medication specifically designed to treat Nonalcoholic Steatohepatitis (NASH), also known as Metabolic Dysfunction-Associated Steatohepatitis (MASH). This condition affects approximately 6 million to 8 million individuals in the United States, characterized by the liver’s inflammation due to excess fat cells. NASH represents an advanced form of Nonalcoholic Fatty Liver Disease (NAFLD). Other associated complications include cirrhosis, liver failure, and liver cancer.
Previously Focused Treatment Approaches
Historically, NASH treatment strategies have revolved around weight loss through lifestyle modifications such as blood sugar control, a well-balanced diet, and regular physical activity.
Game Changer: Madrigal Pharmaceuticals’ Resmetirom (Rezdiffra)
Madrigal Pharmaceuticals’ new medication, resmetirom, will now be marketed under the brand name Rezdiffra. This groundbreaking treatment gained the FDA’s Breakthrough Therapy designation in April 2022, enabling a faster development and review process for drugs intended to address serious conditions with substantial evidence of improvement compared to currently available therapies.
Mechanism of Action and Administration
Rezdiffra is a first-in-class thyroid hormone receptor agonist. It acts on the liver to help minimize fat accumulation by activating this specific receptor. This oral medication is prescribed for individuals diagnosed with NASH and moderate to advanced fibrosis, making it an essential addition to a healthy diet and exercise regimen.
Clinical Trial Results
Results from clinical trials, published in February 2023, revealed that over 25% of participants taking an 80mg dosage achieved NASH resolution without worsening fibrosis. Approximately 30% of those receiving a 100mg dose and less than 10% of placebo recipients experienced similar benefits. Furthermore, the medication showed significant improvements in decreasing LDL or “bad” cholesterol levels and liver enzyme levels.
Adverse Events and Interactions
The majority of adverse events reported during the trial were mild or moderate, with diarrhea and nausea being the most common. Rezdiffra may interact significantly with certain medications, specifically statins used for cholesterol reduction. The FDA advises careful monitoring and consultation between healthcare providers and patients when combining these drugs.
Accelerated Approval and Future Steps
The FDA granted Rezdiffra accelerated approval. Madrigal is now required to conduct a post-approval study to further verify the drug’s clinical benefit.
A New Era for NASH Community
Wayne Eskridge, CEO of the Fatty Liver Foundation, commented on this milestone in a Madrigal press release, expressing optimism for the NASH community:
“I believe this approval milestone will bring new energy and momentum to the NASH community, accelerating our efforts to improve disease education, build care pathways, and expand investment in NASH research.”
Availability and Assistance Programs
Madrigal anticipates that Rezdiffra will become available next month. They have also established an assistance program to help those without insurance access the medication.
