3D Illustration of a human liver organ.
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Good morning! Hundreds of thousands of Americans with a standard and potentially life-threatening type 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,” for use together with weight loss plan and exercise. The corporate expects the medication to be available next month with a hefty price tag of $47,400 per yr before insurance and other rebates.
So, why is that this approval so necessary?
Initially, people affected by the disease badly need a treatment. Rezdiffra is specifically approved to treat patients with nonalcoholic steatohepatitis, or NASH, who even have moderate-to-advanced liver scarring.
Some specialists have began calling the condition metabolic dysfunction-associated steatohepatitis, or MASH, to avoid potentially stigmatizing language.
MASH is a serious type of liver disease characterised by excess fat buildup and inflammation. It will probably result in liver scarring and in additional severe cases, liver failure or cancer. The condition is usually related to other health problems, resembling hypertension, Type 2 diabetes and obesity.
Roughly 6 million to eight million people within the U.S. have MASH with moderate-to-advanced liver scarring, based on an estimate cited by the FDA.
The agency’s approval can also be a giant deal since it means Madrigal has succeeded in an area where several other drugmakers have failed – or are still attempting to crack.
That offers Madrigal an edge in a market that may very well be huge: The MASH industry may very well be value nearly $26 billion by 2032 across the U.S., France, Germany, Italy, Spain, the U.K. and Japan, based on 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 necessary detail within the approval that might bode well for the complete MASH space: The FDA is not requiring patients to get a liver biopsy to find out their eligibility for Madrigal’s drug.
That refers to a procedure through which a physician removes a small piece of liver tissue from a patient so that they can examine it under a microscope for signs of harm or disease.
“The absence of liver biopsy requirement in Rezdiffra’s label should buoy biotech stocks” of firms developing MASH treatments and “potentially speed up and broaden patient access,” William Blair analyst Andy Hsieh wrote in a note Friday.
On this photo illustration, the Madrigal Pharmaceuticals logo is displayed on a smartphone screen.
Rafael Henrique | SOPA Images | Lightrocket | Getty Images
There are a handful of biotech firms with promising experimental MASH treatments:
However the query for all of those firms is how much a blockbuster class of medication called GLP-1s will dominate the MASH market.
GLP-1s work by mimicking a hormone produced within the gut to suppress an individual’s appetite and regulate blood sugar.
They include Novo Nordisk‘s weight reduction drug Wegovy and diabetes treatment Ozempic. Eli Lilly‘s respective weight reduction and diabetes injections Zepbound and Mounjaro are also a part of the category, but goal a further hormone called GIP.
- Novo Nordisk is studying semaglutide, also referred to as Wegovy and Ozempic, in a late-stage trial for treating MASH.
- Eli Lilly released positive mid-stage data on tirzepatide, also referred to as Zepbound and Mounjaro, in treating MASH, and expects to present full data from that trial later this yr.
- Zealand Pharma and Boehringer Ingelheim released positive mid-stage data on an experimental drug called survodutide in MASH patients. The businesses are also studying the treatment in late-stage trials as a treatment for obesity. The treatment prompts GLP-1 and one other hormone called glucagon.
- Merck is studying a drug called efinopegdutide in mid-stage trials. The treatment prompts GLP-1 and one other hormone called glucagon.
Mizuho healthcare equity strategist Jared Holz wrote in an email to investors Friday that GLP-1 uptake is one factor that can be “key regarding the broader NASH thesis.”
He said that is because “there isn’t a doubt in our minds that using the weight-loss therapies will greatly reduce liver fat and would expect many doctors (a minimum of primary care) to go for GLPs first, before referring patients to specialists.”
Wegovy, Ozempic, Zepbound and Mounjaro are already pricey at around $1,000 monthly, or around $13,000 annually, before insurance and other rebates. But that is still far lower than the nearly $50,000 annual price tag of Madrigal’s drug.
We’ll likely have a greater idea of what the MASH drug landscape will seem like over the following yr – and the way big a threat GLP-1s can be – so stay tuned for our coverage!
Be at liberty to send any suggestions, suggestions, story ideas and data to Annika at annikakim.constantino@nbcuni.com.
Latest in health-care technology
Takeaways from the HIMSS conference
Attendees at HIMSS in Orlando, Florida 2024.
Courtesy of HIMSS
Perhaps unsurprisingly, generative artificial intelligence dominated my conversations on the HIMSS conference in Orlando, Florida last week.
As I raced across the exhibition floor to fulfill with executives from firms like Microsoft, Google, Epic Systems, GE HealthCare, Salesforce and more, the subject was quite literally hard to avoid. I walked by a random sample of around 40 vendors on the conference, as an example, and about 75% of them had the word “AI” displayed prominently on their booths.
AI’s popularity at HIMSS was expected. As I discussed in last week’s newsletter, it stole the show eventually yr’s conference, too. But this yr, the conversation across the role of AI in health care had matured. As an alternative of discussing long-term, far-reaching applications of the technology, most of my conversations were about the way it is being tested and utilized in practice.
The most well-liked use case by far was ambient clinical documentation, which got here up over and another time. Ambient clinical documentation tools allow doctors to consensually record their visits with patients, and people conversations are mechanically transformed into clinical notes and summaries using AI.
Corporations like Microsoft’s Nuance Communications, Abridge and Suki have developed these capabilities, which they argue will help reduce doctors’ administrative workloads and prioritize meaningful connections with patients.
Administrative tasks are a serious problem for doctors across the U.S. health-care system, as they’re one in every of the first drivers of burnout, based on a survey published by Athenahealth in February. Because of this, health systems have plenty of interest in tools like ambient clinical documentation that might help reduce this clerical burden.
Organizations like Stanford Health Care and California-based UCI Health announced at HIMSS that they’re rolling out ambient clinical documentation tools across their entire enterprises. Since automating documentation does in a roundabout way impact patients’ diagnoses or care, I expect we’ll see a number of similar announcements across the country this yr. It’s a better, less dangerous way for health systems to deploy generative AI at scale.
Lots of the firms I spoke with are dipping their toes into more patient-facing and diagnostic AI applications, but I feel it’ll be some time before we see doctors and nurses widely using the technology to support their treatment decisions.
Be at liberty to send any suggestions, suggestions, story ideas and data to Ashley at ashley.capoot@nbcuni.com.