FDA Approves First-Ever Treatment for Common and Deadly Liver Disease NASH

On Thursday, 14 March, the Food and Drug Administration (FDA) made history by approving the first-ever treatment for nonalcoholic steatohepatitis (NASH), a common and potentially fatal form of liver disease that affects millions globally. This decision marks a significant milestone in the medical field and offers hope to patients suffering from this debilitating condition.

Madrigal Pharmaceuticals' Success

The approval of Madrigal Pharmaceuticals' drug, to be marketed as Rezdiffra, is a major breakthrough in the battle against NASH. It is worth noting that larger pharmaceutical companies have previously encountered difficulties in developing a successful treatment for this disease, making Madrigal's accomplishment even more remarkable.

Impact on the Market

Following the FDA's announcement, shares of Madrigal Pharmaceuticals surged by more than 20% in extended trading on Thursday. This significant increase underscores the market's positive response to the approval of the groundbreaking medication.

Characteristics of NASH and Approved Treatment

NASH is a severe liver disease characterized by the accumulation of excess fat and inflammation in the liver, which can progress to liver scarring, failure, and even cancer. The newly approved drug, Rezdiffra, is specifically intended for patients with NASH who have moderate-to-advanced liver scarring. It is crucial to emphasize that the treatment should be complemented with a suitable diet and exercise regimen, as stipulated by the FDA.

Prevalence and Availability of the Drug

An estimated 6 to 8 million individuals in the U.S. are affected by NASH with moderate-to-advanced liver scarring, as cited by the FDA. Madrigal Pharmaceuticals has announced that Rezdiffra will be available as early as April, with the company also establishing an assistance program to facilitate access to the medication for uninsured individuals.

FDA's Perspective and Accelerated Approval

Dr. Nikolay Nikolov, acting director of the FDA's Office of Immunology and Inflammation, highlighted the significance of the new treatment, emphasizing that patients with NASH and liver scarring previously lacked a direct means of addressing their liver damage. The FDA granted Rezdiffra an "accelerated approval," a designation reserved for drugs that fulfill an unmet medical need for serious conditions. This allows for expedited approval while necessitating further study to validate the clinical benefits of the treatment.

Mechanism of Action and Clinical Efficacy

Rezdiffra operates by activating a thyroid hormone receptor in the liver, which aids in reducing fat accumulation. Administered orally on a daily basis, the medication demonstrated positive results in a late-stage study, effectively alleviating NASH symptoms and improving liver scarring without exacerbating the condition. Moreover, the incidence of serious adverse events was comparable between the group receiving the drug and the placebo group, indicating a favorable safety profile.

Side Effects and Naming Consideration

Common side effects related to the treatment included diarrhea, nausea, and vomiting. Interestingly, there is a growing trend among specialists to refer to NASH as metabolic dysfunction-associated steatohepatitis (MASH) to avoid potential stigma associated with the condition.