EMA Recommends Rezdiffra for MASH Liver Fibrosis
The European Medicines Agency (EMA) has recommended conditional marketing authorization for Rezdiffra (resmetirom) for adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced liver fibrosis.
MASH was formerly known as nonalcoholic fatty liver disease (NASH). There are currently no authorized treatments for MASH in the EU, making Rezdiffra a potential first.
MASH is a serious liver disease that occurs when fat accumulates in the liver, causing inflammation. Left untreated, it can lead to cirrhosis and cancer. Symptoms may only present in more advanced stages of the condition, at which point they may include discomfort or pain in the upper right abdomen, muscle weakness or loss, and swelling in the abdomen or legs.
Estimates suggest that up to 5% of people in Europe have MASH and that around 25% have metabolic dysfunction‐associated steatotic liver disease (MASLD), the condition that precedes MASH. MASLD was previously known as nonalcoholic fatty liver disease (NAFLD).
The active substance of Rezdiffra is resmetirom, which is a partial agonist of the thyroid hormone receptor-beta. It works by promoting lipophagy and hepatic fatty acid beta-oxidation to reduce liver fat, inflammation, and liver fibrosis.
The EMA's decision comes after interim results from a pivotal, ongoing phase 3 trial including 966 adults with biopsy-confirmed MASH with varying stages of fibrosis. Patients were randomly assigned treatment on a 1:1:1 ratio to receive once-daily resmetirom at 80 mg or 100 mg, or a placebo.
The primary endpoints were MASH resolution at week 52 with no worsening of fibrosis, and a reduction in fibrosis by at least one stage alongside no worsening of NAFLD activity score. MASH resolution included a reduction in the NAFLD activity score by ≥ 2 points. Scores range from 0 to 8; higher scores indicate more severe disease.
After 12 months, 30% of patients in the 100 mg resmetirom group and 26% of those in the 80 mg group achieved MASH resolution with no worsening fibrosis compared with 10% in the placebo group.
Meanwhile, 26% of patients in the 100 mg resmetirom group and 24% of patients in the 80 mg group experienced fibrosis improvement by at least one stage with no worsening of NAFLD activity score compared with 14% in the placebo group.
The most frequent side effects were diarrhea, nausea, itching, and pruritus. Diarrhea and nausea were more frequent among those taking resmetirom than those taking the placebo. The rate of serious adverse events was similar across all groups and ranged from 10.9% to 12.7%.
Rezdiffra will be available as 60 mg, 80 mg, and 100 mg film-coated tablets. It should be taken alongside diet and exercise.
The opinion adopted by the EMA will now go to the European Commission to await a decision on EU-wide marketing authorization.
In the meantime, the EMA has required Rezdiffra's applicant to complete both the pivotal and another ongoing trial to provide further data regarding the drug's efficacy. This conditional approval comes as the EMA perceives that the benefits to patients from immediate availability outweigh the risk inherent in incomplete data.
Detailed recommendations for using Rezdiffra will be described in the summary of product characteristics, which will be published on the EMA website in all official European Union languages.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
New York Times
an hour ago
- New York Times
Is It Wrong to Push Ozempic on My Spouse?
My husband's lifestyle is very sedentary, with minimal exercise (20 to 60 minutes a week), though he does eat a largely healthy diet. I've watched him struggle with his weight over the 15 years we've been together, cycling through various approaches to diet and exercise. Even when he succeeds in losing weight, it always comes back, and now he seems to have given up. As we both approach 50, I worry that his B.M.I., which is 30, and his B.R.I. (body roundness index, a measure of abdominal fat) are high (he can't even button some of his shirts around the middle), which could lead to other health issues. I've already tried encouraging him to move more and eat better, but I can't schedule every one of my workouts for us to exercise together, and he dislikes some of the routines I do, anyway. He's also very sensitive about his weight. Is it wrong for me to try to get him to take Ozempic? I'm hoping that losing weight will help boost his energy levels, which might lead to more self-care. I know it's not my body, and I'm not his doctor, but as his wife I also know it will fall to me to care for him if health issues arise. — Name Withheld From the Ethicist: It's natural to worry about the health of someone you love — especially as the years accumulate and the lines between caring for yourself and caring for your partner begin to blur. Long-term relationships involve a particular kind of vigilance: You notice the wince, the shortened breath, the unbuttoned shirt. This isn't control; it's love in the form of everyday attention. Obesity really is correlated with health problems, and if your husband could lose weight sustainably, his life might improve in ways that matter. Historically, weight loss has been a story of perpetual disappointment — diets abandoned, exercise routines that peter out, New Year's resolutions that start wobbling the following evening when it becomes clear that celery sticks are an imperfect substitute for chocolates. GLP-1 drugs, such as Ozempic, have helped many people break that cycle. Still, even sound advice can land wrong if it touches a nerve. If your husband is sensitive about his weight, the real challenge isn't knowing what's best; it's finding a way to talk about it that feels loving, not critical. Try to keep the focus on the prospect of his feeling fitter and on your hope for more good years ahead. Maybe suggest he talk to his doctor — not as a directive but as a way to explore options. You're not pushing him to 'fix' himself; you're looking out for him. If it turns out to be a difficult conversation all the same? Loving someone can mean risking that discomfort — for the sake of all the days you haven't yet lived together. Want all of The Times? Subscribe.
Wall Street Journal
an hour ago
- Wall Street Journal
Three Siblings, One Fatal Gene: A Family's Fight Against Early-Onset Alzheimer's
Hannah Richardson is hopeful about her future and its endless possibilities. But the 24-year-old's plans are clouded by an unthinkable reality—there is a 50% chance she will develop Alzheimer's disease in her 30s. Hannah's family has a history of a rare genetic mutation that, when inherited, virtually guarantees that the carrier will die of an aggressive form of Alzheimer's early in life. No drug has been found to stop it. But now researchers are exploring a new avenue: Could pre-emptive treatment slow or even halt the memory-robbing disease in people at high risk of developing it?
Yahoo
2 hours ago
- Yahoo
Plans unveiled for NHS funding to be linked to patient feedback
NHS funding could be linked to patient feedback under new plans, with poorly performing services that "don't listen" penalised with less money. As part of the "10 Year Health Plan" to be unveiled next week, a new scheme will be trialled that will see patients asked to rate the service they received - and if they feel it should get a funding boost or not. It will be introduced first for services that have a track record of very poor performance and where there is evidence of patients "not being listened to", the government said. This will create a "powerful incentive for services to listen to feedback and improve patients' experience", it added. Sky News understands that it will not mean bonuses or pay increases for the best performing staff. NHS payment mechanisms will also be reformed to reward services that keep patients out of hospital as part of a new 'Year of Care Payments' initiative and the government's wider plan for change. Speaking to The Times, chief executive of the NHS Confederation Matthew Taylor expressed concerns about the trial. He told the newspaper: "Patient experience is determined by far more than their individual interaction with the clinician and so, unless this is very carefully designed and evaluated, there is a risk that providers could be penalised for more systemic issues, such as constraints around staffing or estates, that are beyond their immediate control to fix." He said that NHS leaders would be keen to "understand more about the proposal", because elements were "concerning". Read more from Sky News Health Secretary Wes Streeting said: "We will reward great patient care, so patient experience and clinical excellence are met with extra cash. These reforms are key to keeping people healthy and out of hospital, and to making the NHS sustainable for the long-term as part of the Plan for Change." In the raft of announcements in the 10 Year Health Plan, the government has said 201 bodies responsible for overseeing and running parts of the NHS in England will be scrapped. These include Healthwatch England, set up in 2012 to speak out on behalf of NHS and social care patients, the National Guardian's Office, created in 2015 to support NHS whistleblowers, and the Health Services Safety Investigations Body (HSSIB). Elsewhere, the new head of NHS England Sir Jim Mackey said key parts of the NHS appear "built to keep the public away because it's an inconvenience". "We've made it really hard, and we've probably all been on the end of it," he told the Daily Telegraph. "The ward clerk only works nine to five, or they're busy doing other stuff; the GP practice scrambles every morning."
