Resmetirom's approval in Europe and the UK: When will the MASH medicine be available?

Metabolic dysfunction-associated steatohepatitis (MASH/NASH) is increasingly recognized as a significant global health issue, particularly due to the rising rates of obesity. Until recently, there were no specific medications approved for this condition. Especially for patients with moderate to severe liver scarring (fibrosis).

The FDA's accelerated approval of Rezdiffra (resmetirom) in 2024 marks a pivotal change. With approximately 44% of Europeans grappling with liver diseases like MASH, timely access to this innovative fatty liver treatment in Europe is crucial ¹.

When will resmetirom receive EMA approval and be available to patients across Europe and the UK? Here's what you need to know.

What are Rezdiffra's uses?

Rezdiffra (resmetirom) is used for the treatment of nonalcoholic steatohepatitis (NASH) in patients who have moderate to advanced liver scarring (fibrosis). NASH is since recently referred to as metabolic dysfunction-associated steatohepatitis (MASH). Rezdiffra is not applicable for patients with cirrhosis of the liver.

The way Rezdiffra works is by activating a thyroid hormone receptor that reduces the accumulation of fat in the liver ².

Can Rezdiffra reverse fibrosis?

According to clinical trial results, Rezdiffra (resmetirom) may improve or reverse fibrosis in about 25% of patients ². This is the first medicine that has reportedly shown an ability to reverse liver scarring.

Rezdiffra's efficacy in clinical trials

The FDA's approval of resmetirom was based on the preliminary results of the ongoing Phase 3 MAESTRO-NASH trial. While the trial will be completed in 2027, some of the key efficacy results that have been reported after 52 weeks of treatment include:

• 26-27% of patients treated with resmetirom achieved NASH resolution or an improvement in liver scarring;
• 24-26% of patients treated with resmetirom improved by at least one stage on the NAFLD activity score;
• Low-density lipoprotein cholesterol levels were reduced by up to 16.3% in the resmetirom group;
• The most common side effects of Rezdiffra were reported to be nausea and diarrhea ³.

These results demonstrate the potential of this new liver disease medicine. And while Rezdiffra has been available in the USA since April 2024, when can patients in Europe and the UK expect to access the treatment?

When will resmetirom get EMA approval?

Resmetirom has been under review by the EMA since March 2024. And while the EMA has indicated the possibility of granting it conditional approval, as of December 2024, that is not the case yet ⁴.

Typical EMA approval timelines can take up to 210 days, but in the case of resmetirom, the process is taking longer. This may be due to additional information being requested from the manufacturer. Hopefully, resmetirom will get its EU approval any day now.

When will resmetirom be available in Europe?

Even if resmetirom gets approved in Europe soon, that unfortunately doesn't mean it will be available immediately. The medicine's manufacturer, Madrigal Pharmaceuticals, is aiming to launch the medicine in Europe in the second half of 2025, pending its EMA approval ⁵. However, some countries will be faster with the launch than others.

Across the EU, Germany tends to be the first country to launch a newly approved medicine - on average, within 133 days. At the other end of the spectrum is Romania, with 899 days on average.

So, if Madrigal starts rolling out its market launch efforts in late 2025, some European patients may already start treatment with resmetirom within 2025. Others, however, may be waiting until 2027 and beyond.

Will resmetirom be approved in the UK?

Most probably, yes, given the status of the medicine as the only treatment for non-alcoholic fatty liver disease with medium to advanced liver fibrosis. However, as of December 2024, Rezdiffra is not yet licensed in the UK, and there is no timeline available for its licensing.

Technically, post-Brexit regulations allow for almost immediate approval of a medicine in the UK after it has been given the green light of another trusted regulator (such as the FDA). It's possible that the MHRA decides to take this road. If it does, Rezdiffra could be approved in the UK very soon.

When will Rezdriffa be available on the NHS?

In order to become available on the NHS, resmetirom needs to receive a positive assessment from NICE. An assessment is officially initiated, but there is no decision date, as the medicine is not yet approved in the UK. In theory, a novel medicine becomes available on the NHS within 3 months of a positive NICE decision.

In the best-case scenario, where Rezdriffa gets MHRA approval by the end of 2024 and NICE makes a decision soon after, it's possible that resmetirom will be available to patients in the UK by mid 2025. However, time will tell if this scenario is realistic.

How to get resmetirom before it's available in your country

While medicine approval timelines are not always encouraging, there is good news. You don't actually have to wait until Rezdiffra gets EMA approval or becomes available in your country. If your doctor believes the medicine can benefit your condition, they can prescribe it to you immediately. And with a prescription, you can buy resmetirom right away.

Buying resmetirom for personal use

Thanks to a regulation known as Named Patient Import, patients around the world can import a medicine before it's approved or available in their country. In order to qualify for this regulation, the medicine in question needs to:

• be indicated for a life-threatening or debilitating disease;
• be prescribed by the patient's treating doctor;
• have approval somewhere in the world;
• have no alternatives in the patient's country.

Would you like to use the Named Patient Import regulation to start your treatment with resmetirom immediately? First talk to your doctor and get a suitable prescription.

Already have a prescription? Share it with our expert team. We'll help you buy Rezdiffra immediately, before its EMA approval or MHRA approval. 

References:

1. Rezdiffra. Drugs.com, Accessed 11 December 2024. 
2. MACMILLAN, CARRIE. FDA Approves Rezdiffra, the First Drug for NASH, a Form of Fatty Liver Disease. Yale Medicine, Accessed 11 December 2024.
3. A Phase 3, Randomized, Controlled Trial of Resmetirom in NASH with Liver Fibrosis. The New England Journal of Medicine, Accessed 11 December 2024. 
4. EMA says it will consider conditional approval for NASH drugs using intermediate endpoints. Raps.org, Accessed 11 December 2024. 
5. ASH: Eli Lilly touts novel BTK trial win for Jaypirca as execs defend survival data blemish. Fierce Pharma, Accessed 11 December 2024.