On Thursday, March 14th local time, the US Food and Drug Administration (FDA) announced on its official website that it has approved the drug Rezdiffra for the treatment of non-alcoholic steatohepatitis (NASH) with moderate to severe liver fibrosis.
Non alcoholic fatty liver disease (NAFLD) is a liver condition in individuals who drink little or no alcohol. Some NAFLD patients may suffer from non-alcoholic steatohepatitis (NASH), a severe fatty liver disease in which fat deposits in the liver, leading to liver swelling and damage.
NASH is often closely related to obesity, diabetes, hyperlipidemia and other metabolic syndrome, which may worsen and may lead to severe liver scar (liver fibrosis), even cirrhosis and liver cancer. This damage is similar to that caused by heavy alcohol use.
The FDA press release mentioned that there are multiple statistical estimates that approximately 6 to 8 million people in the United States suffer from NASH with moderate to severe liver scars, and this number is expected to continue to increase.
The drug Rezdiffra can activate thyroid hormone receptors in the liver, thereby promoting fat breakdown metabolism and reducing fat accumulation.
FDA First Approval
Previously, Rezdiffra, which was taken orally once a day, was qualified for breakthrough therapy, fast track, and priority review by the FDA, and successfully passed the "accelerated approval" process on Thursday, becoming the "first in class" drug in this field.
Prior to this, NASH's treatment plan focused on lifestyle changes such as healthy eating and moderate exercise. FDA senior official Nikolay Nikolov wrote that Rezdiffra's approval will provide patients with treatment options other than diet and exercise for the first time. The institution emphasizes that drug therapy still needs to be combined with diet and exercise.
In terms of efficacy, as the basis for FDA approval, a phase 3 clinical trial of Rezdiffra lasting 54 months is still ongoing. The biopsy evaluation in the first 52 weeks of this clinical trial showed that 25.9% and 29.9% of patients in the 80mg and 100mg drug groups achieved NASH symptom relief and liver fibrosis did not worsen, respectively, compared to 9.7% in the placebo group.
In addition, 24.2% and 25.9% of patients in the drug group achieved at least one stage of improvement in fibrosis, and the NAFLD activity score did not deteriorate, respectively. The proportion in the placebo group was only 14.2%, which was the first phase 3 clinical trial to achieve the two main endpoints proposed by the FDA in NASH patients.
Or it may open up another billion dollar market
Media analysis suggests that this drug is expected to open up a market worth billions of dollars. According to research firm Vision Research Reports, the field is expected to become a global market of over $16 billion by 2030. The company also mentioned that many other pharmaceutical companies are also focusing on this field and have not yet achieved breakthroughs.
Investment bank Evercore ISI analyst Liisa Bayko estimates that Rezdiffra's peak annual sales will exceed $5 billion. Benefiting from this news, research and development company Madrid Pharmaceuticals rose more than 20% in the early trading session, and its stock price is expected to reach a new high since May last year.
Leerink Partners analyst Thomas J. Smith also mentioned that Rezdiffra covers a wide range of patient populations and labels indicate that liver biopsy is not required to diagnose the patient's disease. William Blair analyst Andy Hsieh also stated that the absence of liver biopsy requirements is indeed a significant advantage.