Latest news with #EMA
Medscape
29 minutes ago
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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.
Medscape
2 hours ago
- Health
- Medscape
EMA Reviews Alcohol Dependency Drug After French Concerns
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced it will review the use of medicines containing sodium oxybate, a drug used in narcolepsy, in people with alcohol dependency. The review comes at the request of the French Medicines Agency and has been initiated under Article 31 of Directive 2001/83/EC. This procedure is used to address concerns over the safety or benefit-risk balance of a medicine or a class of medicines; to resolve disagreements between member states on issues related to the authorization of medicines; or to give an opinion on an issue of Europe-wide interest so that the agency can make a recommendation for a harmonized position across the EU. The European Commission, any member state, or the company that markets the medicine can initiate a referral. In a referral, the European Medicines Agency (EMA) is requested to conduct a scientific assessment of a particular medicine or class of medicines on behalf of the European Union. It involves the matter being referred to the Pharmacovigilance Risk Assessment Committee (PRAC) after due notice to the EMA, all member states, and the European Commission. Narcolepsy Indication Sodium oxybate (Xyrem) is the sodium salt of gamma-hydroxybutyric acid. As an oral solution (500 mg/mL), it is indicated for the treatment of narcolepsy with cataplexy in adult patients, adolescents, and children from the age of 7 years. The drug is a central nervous system depressant believed to act by attaching to brain cell receptor molecules to promote delta (slow) brain waves and nighttime sleep. When taken at bedtime, Xyrem increases sleep depth and length, reducing the number of sleeping periods during the day and improving symptoms of narcolepsy. Because of the drug's potential for abuse, it can only be obtained with a special prescription, and treatment should be initiated and maintained under the guidance of a doctor with experience in treating sleep disorders. Doctors are advised to check for a history of or susceptibility to drug abuse before treatment, and to monitor for misuse and abuse during treatment. Approved for Alcohol Abuse in Austria and Italy The drug has also been suggested to reduce pain and improve function in fibromyalgia, but the EMA said the data were insufficient to support its use for this in the European population. However, sodium oxybate is also approved in Italy and in Austria, under the name Alcover, to treat alcohol dependence and alcohol withdrawal syndrome. It is this use that the review will examine. The EMA has confirmed the positive benefit-risk profile of the treatment as prescribed in Italy and Austria. France, however, despite having a high prevalence of alcohol problems, has resisted its use for this purpose. Trials have suggested that sodium oxybate can rapidly alleviate symptoms of alcohol withdrawal syndrome and was at least as effective as diazepam and clomethiazole. It can also be used in the maintenance of abstinence in alcohol-dependent patients, in whom it is reportedly as effective as naltrexone or disulfiram. Because existing approved medications are only modestly effective, there is a pressing need for effective treatments with an alternative mode of action, particularly in patients with very high consumption levels. Sodium oxybate also has a favorable safety profile. However, studies in alcohol use disorder have been limited and further investigations have been recommended.
Medscape
2 hours ago
- Health
- Medscape
Darzalex Monotherapy OK'd for Smoldering Multiple Myeloma
At its June 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended extending the indications for Darzalex (daratumumab) solution for injection as monotherapy to the treatment of adult patients with smoldering multiple myeloma at high risk of developing multiple myeloma. Darzalex is a monoclonal antibody used to treat adults with multiple myeloma and light chain amyloidosis. Good Response to Treatment Daratumumab attaches to the CD38 protein, found in large amounts on abnormal white blood cells in multiple myeloma and light chain amyloidosis. By attaching to CD38 on these cells, daratumumab activates the immune system to kill the abnormal white blood cells. Darzalex as monotherapy was investigated in two main studies involving multiple myeloma patients whose disease relapsed after, or was refractory to, at least two previous treatments, including a proteasome inhibitor and an immunomodulatory medicine. Response to treatment was measured by the disappearance of, or at least a 50% reduction in, protein produced by multiple myeloma cells. In the first study, around 29% of the patients who received daratumumab responded to treatment. In the second study, 36% of patients responded. In these studies, daratumumab was not compared with any other treatment. Full Indications The CHMP highlighted that the full indications for Darzalex solution for injection for will now be: For multiple myeloma: In combination with lenalidomide and dexamethasone or with bortezomib, melphalan, and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant In combination with bortezomib, lenalidomide, and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma In combination with bortezomib, thalidomide, and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant In combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy In combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received one prior therapy containing a proteasome inhibitor and lenalidomide and were lenalidomide-refractory, or who have received at least two prior therapies that included lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or after the last therapy As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy For smoldering multiple myeloma: As monotherapy for the treatment of adult patients with smoldering multiple myeloma at high risk of developing multiple myeloma For light chain (AL) amyloidosis: In combination with cyclophosphamide, bortezomib, and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain amyloidosis The indications for Darzalex concentrate for solution for infusion remained unchanged, the CHMP said. Rob Hicks is a retired UK National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications.
Medscape
3 hours ago
- Health
- Medscape
Europe Recommends Stem Cell Therapy for Blood Cancers
At its June 2025 meeting, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a recommendation for conditional marketing authorization in the European Union for Zemcelpro (dorocubicel/allogeneic umbilical cord-derived CD34- cells non-expanded, Cordex Biologics International Limited) to treat adults with hematologic malignancies. A conditional marketing authorization is granted to a medicinal product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required. Hematologic malignancies include leukemias, lymphomas, myelodysplastic syndrome, and myelomas. The only potential curative treatment option for several of these cancers is allogeneic hematopoietic stem cell transplantation (allo-HSCT). This type of transplant involves using donated stem cells to replace the recipient's bone marrow cells to form new bone marrow that produces healthy blood cells. Zemcelpro can be used in patients requiring an allo-HSCT following myeloablative conditioning — chemotherapy and/or radiotherapy — for whom no other type of suitable donor cells is available, the agency said. Novel Cell Therapy Zemcelpro is a novel cell therapy containing expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. By increasing the number of cells, Zemcelpro makes the stem cells from a small cord blood unit more effective. The benefit of Zemcelpro is its ability to induce neutrophil and platelet engraftment, as observed in two single-arm, open-label, phase 2 clinical studies. The decision by the CHMP was largely based on a pooled analysis of these studies, which included 25 patients. In total, 84% of patients achieved neutrophil engraftment within a median time of 20 days, and 68% of patients achieved platelet engraftment within a median time of 40 days. In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), EMA's expert committee for cell- and gene-based medicines, found that the benefits of Zemcelpro outweighed the possible risks in patients with hematologic malignancies requiring allo-HSCT for whom no matched donor cells were available. Further Study Results Requested Zemcelpro will be available as a ≥ 0.23 x 106 viable CD34+ cells/mL / ≥ 0.53 x 106 viable CD3+ cells/mL dispersion for infusion. The most common side effects with the treatment include lymphopenia, infections, anemia, neutropenia, thrombocytopenia, leukopenia, hypogammaglobulinemia, febrile neutropenia, hypertension, engraftment syndrome, pneumonia, and graft-vs-host disease (GvHD). Zemcelpro was supported through EMA's Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs. To confirm the safety and efficacy of the treatment, the company has been requested to submit long-term follow-up results of the single-arm studies, and conduct a randomized controlled study as well as a study based on a patient registry.
Medscape
3 hours ago
- Health
- Medscape
HIV Prevention Vaginal Ring Recommended for 16-Year-Olds
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended an extension of indication for dapivirine vaginal ring 25 mg to include its use in women from 16 years of age. The EMA said there had been some uncertainties about the risk for resistance and the benefits in younger women, which needed to be addressed. Upon further study, the ring was shown to be moderately effective at lowering the risk for HIV-1 infection following vaginal intercourse, with its side effects considered manageable. As such, the EMA decided that its benefits outweigh the risks in women who are not using oral pre-exposure prophylaxis (PrEP). The dapivirine ring was originally approved in July 2020 to reduce the risk for HIV-1 infection in women 18 years and older through vaginal intercourse, when taking PrEP medicines by mouth was not feasible. It is the first long-acting technology approved for HIV prevention in women and is intended for use outside the EU. The device was submitted under a regulatory procedure, EU- Medicines for all (EU-M4All) that enables the EMA, in collaboration with the World Health Organization (WHO), to provide scientific opinions on high priority human medicines that are intended for markets outside the EU. The process allows the European agency to support global regulatory capacity building and contributes to the protection and promotion of public health beyond the EU. Its overall aim is to facilitate access to essential medicines in low- and middle-income countries that are intended to prevent or treat diseases of major public health interest. The flexible silicone ring is inserted in the vagina for a period of 28 days, after which it should be replaced by a new one. It works by slowly releasing dapivirine over the course of a month, which blocks the ability of HIV to make copies of itself inside healthy cells. It can be inserted and removed by the user. A study conducted in almost 2000 women in sub-Saharan Africa showed that dapirivine vaginal ring 25 mg lowered the chances of being infected with HIV by more than one third over the course of 24 months compared with the placebo group. It was first developed by the International Partnership for Microbicides and since acquired by the Population Council in 2022. It was first approved in 2021 by the Medicines Control Authority of Zimbabwe and has since been approved in several additional African countries. The ring has been on the WHO's prequalification list of medicines since November 2020 following positive scientific opinion from the EMA. The WHO has said it is intended to reduce the risk of acquiring HIV during vaginal sex for women who are at substantial HIV risk as a complementary prevention approach in addition to other safer-sex practices. The CHMP's opinion to extend the indication for this medicine to include its use in women from 16 years of age expands the HIV prevention choice beyond condoms and oral PrEP and could be an important tool to protect younger women from becoming HIV-positive.
