Healthy Returns: The first drug for a common, deadly liver disease is here – and more are coming

The introduction of the first drug for a prevalent and life-threatening liver disease marks a significant milestone in healthcare. With more treatments in development, there's hope for improved outcomes for patients. This development underscores the progress in medical research and the potential for transformative therapies to address previously unmet medical needs, offering new avenues for managing and treating liver diseases more effectively.

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Good morning! Millions of Americans with a common and potentially life-threatening form of liver disease will soon have access to the first-ever treatment for the condition. 

In a landmark decision on Thursday, the Food and Drug Administration approved Madrigal Pharmaceuticals

’ drug “Rezdiffra,” to be used along with diet and exercise. The company expects the medicine to be available next month with a hefty price tag of $47,400 per year before insurance and other rebates. 

So, why is this approval so important? 

First of all, people suffering from the disease badly need a treatment. Rezdiffra is specifically approved to treat patients with nonalcoholic steatohepatitis, or NASH, who also have moderate-to-advanced liver scarring.

Some specialists have started calling the condition metabolic dysfunction-associated steatohepatitis, or MASH, to avoid potentially stigmatizing language.

MASH is a serious form of liver disease characterized by excess fat buildup and inflammation. It can lead to liver scarring and in more severe cases, liver failure or cancer. The condition is often associated with other health problems, such as high blood pressure, Type 2 diabetes and obesity.

Roughly 6 million to 8 million people in the U.S. have MASH with moderate-to-advanced liver scarring, according to an estimate cited by the FDA. 

The agency’s approval is also a big deal because it means Madrigal has succeeded in an area where several other drugmakers have failed – or are still trying to crack. 

That gives Madrigal an edge in a market that could be huge: The MASH industry could be worth nearly $26 billion by 2032 across the U.S., France, Germany, Italy, Spain, the U.K. and Japan, according to an estimate from data analytics firm GlobalData. 

Madrigal’s drug is setting the bar for efficacy and safety for MASH treatment, while also opening the door for medicines still in development. 

There’s one important detail in the approval that could bode well for the entire MASH space: The FDA isn’t requiring patients to get a liver biopsy to determine their eligibility for Madrigal’s drug. 

That refers to a procedure in which a doctor removes a small piece of liver tissue from a patient so they can examine it under a microscope for signs of damage or disease. 

There are a handful of biotech companies with promising experimental MASH treatments: 

• Akero Therapeutics
•  released positive mid-stage trial data on its drug efruxifermin earlier this month and is studying the treatment in late-stage trials. 
• Viking Therapeutics
•  is studying a drug called VK2809 in mid-stage trials, with 52-week biopsy data expected in the first half of the year. 
• 89bio
•  is studying and planning late-stage trials on its drug pegozafermin. 
• Aligos Therapeutics
•  is studying a drug called ALG-055009 in mid-stage trials. 

But the question for all of those companies is how much a blockbuster class of drugs called GLP-1s will dominate the MASH market. 

GLP-1s work by mimicking a hormone produced in the gut to suppress a person’s appetite and regulate blood sugar. 

They include Novo Nordisk

’s weight loss drug Wegovy and diabetes treatment Ozempic. Eli Lilly

’s respective weight loss and diabetes injections Zepbound and Mounjaro are also part of the class, but target an additional hormone called GIP. 

• Novo Nordisk is studying semaglutide, also known as Wegovy and Ozempic, in a late-stage trial for treating MASH. 
• Eli Lilly released positive mid-stage data on tirzepatide, also known as Zepbound and Mounjaro, in treating MASH, and expects to present full data from that trial later this year. 
• Zealand Pharma and Boehringer Ingelheim released positive mid-stage data on an experimental drug called survodutide in MASH patients. The companies are also studying the treatment in late-stage trials as a treatment for obesity. The treatment activates GLP-1 and another hormone called glucagon. 
• Merck
•  is studying a drug called efinopegdutide in mid-stage trials. The treatment activates GLP-1 and another hormone called glucagon. 

Mizuho healthcare equity strategist Jared Holz wrote in an email to investors Friday that GLP-1 uptake is one factor that will be “key regarding the broader NASH thesis.” 

He said that’s because “there is no doubt in our minds that the use of the weight-loss therapies will greatly reduce liver fat and would expect many doctors (at least primary care) to opt for GLPs first, before referring patients to specialists.”

Wegovy, Ozempic, Zepbound and Mounjaro are already pricey at around $1,000 per month, or around $13,000 annually, before insurance and other rebates. But that’s still far less than the nearly $50,000 annual price tag of Madrigal’s drug. 

We’ll likely have a better idea of what the MASH drug landscape will look like over the next year – and how big a threat GLP-1s will be – so stay tuned for our coverage!

Feel free to send any tips, suggestions, story ideas and data to Annika at annikakim.constantino@nbcuni.com.

Source: CNBC‍