EMA Recommends New Treatments for MASH, Blood Cancers, and other Conditions
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The European Medicines agency (EMA) has issued recommendations for several new medications, offering potential advancements in treating liver disease, blood cancers, and other conditions.
BRUSSELS – 2025/06/20 13:35:05 – The European Medicines Agency (EMA) has recommended granting marketing authorization for ‘rezdiffra’ (resmerom) within the European Union. This medication is intended for adult patients suffering from non-cirrhotic steatohepatitis associated with metabolic dysfunction (MASH) who have moderate too advanced liver fibrosis.
MASH is characterized by fat accumulation in the liver, leading to inflammation and potential progression to cirrhosis and cancer. Currently, there are no authorized treatments for MASH available in the EU. ‘Rezdiffra’ contains resmemarom, which targets a thyroid hormone receptor in the liver to reduce fat accumulation, inflammation, and fibrosis.
The EMA’s Commitee for Human Medicinal Products (CHMP) based its positive opinion on data from a randomized, controlled clinical trial.The results showed that ‘Rezdiffra’ led to the resolution of MASH without worsening of fibrosis in a significant percentage of patients, compared to a placebo. Specifically, 30 percent of patients receiving 100 mg of resmemarom and 26 percent of those receiving 80 mg achieved resolution, versus 10 percent in the placebo group.
The CHMP has requested that the applicant, Madrigal Pharmaceuticals, complete ongoing trials to provide further data on the effectiveness of ‘Rezdiffra’. Common side effects reported during the trials included diarrhea, nausea, and itching. The recommendation for conditional marketing authorization reflects the EMA’s commitment to providing early access to medicines that address unmet medical needs.
The CHMP’s opinion is a step towards making ‘Rezdiffra’ available to patients.The European Commission will now review the recommendation and make a final decision on marketing authorization. Following approval, individual member states will determine pricing and reimbursement within their national health systems.
The recommendation for conditional marketing authorization reflects the EMA’s commitment to providing early access to medicines that address unmet medical needs.
Other Recommended Medicines
The CHMP also recommended approval for 12 other medications, including:
- ‘Austedo’ (deutetrabenazine) for adults with tardive dyskinesia.
- ‘IMREPLYS’ (sargramostim) for acute radiation hematopoietic syndrome.
- ‘Ogsiveo’ (nirogacestat) for progressive desmoid tumors.
- ‘Zemcelpro’ (non-expanded umbilical cord cells), a stem cell therapy for hematological neoplasms, offering an option for blood cancer patients without an adequate donor. this received support from the EMA’s Priority Medicines (Prime) program.
- Six biosimilar medications: ‘Mynzepli’ (aflibercept), ‘AFIVER’ (aflibercept), ‘VGENFLI’ (aflibercept), and their duplicates.
- ‘Usymro’ (ustekinumab) for plaque psoriasis, psoriatic arthritis, and Crohn’s disease.
- ‘Vivlipeg’ (pegfilgrastim) to reduce neutropenia after chemotherapy.
- Two generic medications: ‘Emtricitabine’ / ‘Rilpivirina’ / ‘Tenofovir alafenamide viatris’ (emtricitabine / rilpivirine / tenofovir alafenamide) for HIV-1.
- ‘Nintenanib viatris’ (ninontenib) for idiopathic pulmonary fibrosis and other interstitial pulmonary diseases.
Frequently Asked Questions
- What is MASH?
- Metabolic dysfunction-associated steatohepatitis (MASH) is a liver disease characterized by fat accumulation, inflammation, and liver cell damage. It can lead to severe complications like cirrhosis and liver cancer.
- What is ‘Rezdiffra’ and how does it work?
- ‘Rezdiffra’ (resmerom) is a medication designed to treat MASH. It works by stimulating a thyroid hormone receptor in the liver, which helps reduce fat accumulation, inflammation, and liver fibrosis.
- What are the potential side effects of ‘Rezdiffra’?
- The most common side effects reported in clinical trials include diarrhea, nausea, and itching.
- Who is ‘Rezdiffra’ intended for?
- ‘Rezdiffra’ is intended for adult patients with non-cirrhotic MASH who have moderate to advanced liver fibrosis.
- What is conditional marketing authorization?
- Conditional marketing authorization is a regulatory mechanism used by the EMA to facilitate early access to medicines that address unmet medical needs, even when complete data is not yet available.
