Evaluation and monitoring of medicines: highlights

Human medicines

Medicines recommended for approval

In 2025, EMA recommended 104 medicines for marketing authorisation, including 38 with new active substances. Of those, the medicines selected in this overview represent significant progress in their therapeutic areas:

• Anktiva, for the treatment of adults with a type of bladder cancer that affects the lining of the bladder (non-muscle invasive bladder cancer, NMIBC) and that is at high risk of growing and spreading. Bladder cancer is one of the most common cancers in the EU, affecting over 200,000 people each year, with most cases being NMIBC.

• Aucatzyl, for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukaemia, a type of cancer of the white blood cells.

• Brinsupri, the first treatment for non-cystic fibrosis bronchiectasis, a serious, chronic, progressive lung disease resulting in damaged airways and severe pulmonary dysfunction, often leading to chronic cough and airflow obstruction due to abnormal mucus production.

• Duvyzat, a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who are still able to walk. DMD is a rare, ultimately lethal genetic disease in which the muscles progressively weaken and lose function. 

• Kisunla, for the treatment of early Alzheimer’s disease. Patients have to be tested to exclude the presence of two copies of the ApoE ε4 gene.

• Rezdiffra, for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), a serious disease where fat cells accumulate in the liver causing chronic inflammation. This is the first authorised treatment for MASH in the EU.

• Teizeild, a first-in-class treatment to delay the onset of stage 3 type 1 diabetes in adults and in children from eight years of age with stage 2 type 1 diabetes.

• Tepezza, for the treatment of adults with moderate to severe Thyroid Eye Disease (TED), also known as Graves’ Eye Disease, a rare autoimmune disease that triggers inflammation of muscles, fat, and other tissues around and behind the eyes. Treatment options for moderate-to-severe TED are limited, most patients are treated with corticosteroids and some patients need multiple reconstructive surgeries.

• Vimkunya, a vaccine to protect young people from 12 years of age and adults against disease caused by the Chikungunya virus.

• Vyjuvek, to treat wounds in patients of all ages with dystrophic epidermolysis bullosa, a serious, ultra-rare genetic skin blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. Vyjuvek is expected to bring substantial therapeutic benefits and improve the quality of life for patients with this skin disorder.

• Waskyra, the first medicine to treat Wiskott-Aldrich syndrome, a rare, inherited disease, seen almost exclusively in males, that affects blood cells and cells of the immune system.

• Yeytuo (lenacapavir), for pre-exposure prophylaxis (PrEP) in combination with safer sex practices to reduce the risk of sexually acquired human immunodeficiency virus type 1 (HIV-1) infection in adults and adolescents at high risk of becoming infected. Yeytuo will facilitate PrEP uptake and compliance because it only has to be administered twice a year via a subcutaneous injection. Lenacapavir was also approved as Lenacapavir Gilead under EU-Medicines for all (EU-M4All).

• Zemcelpro (lenacapavir), a stem cell therapy to treat patients with haematological malignancies (blood cancers) who need a blood stem cell transplant but have no suitable donor.

Early access to medicines that address public health needs

In 2025, three medicines received a recommendation for marketing authorisation following an accelerated assessment: Brinsupri, Vimkunya and Yeytuo . This mechanism is reserved for medicines that address unmet medical needs. It allows for faster assessment of eligible medicines by EMA’s scientific committees (within a maximum of 150 days rather than 210 days).

Eight medicines received a recommendation for a conditional marketing authorisation , one of the possibilities in the EU to give patients early access to new medicines: Anktiva, Aucatzyl, Duvyzat, Ezmekly, Lynozyfic, Rezdiffra, Zemcelpro and Ziihera. The conditional authorisation allows for early approval on the basis of less complete clinical data thann ormally required, because the benefit of earlier patient access outweighs the potential risks of limited data. Marketing authorisation holders for these medicines are subject to specific post-authorisation obligations to generate complete data on the products.

Two medicines (Imreplys and Maapliv) were authorised under exceptional circumstances, a route that allows patients access to medicines that cannot be approved under a standard authorisation as comprehensive data cannot be obtained, either because there are only very few patients with the disease, or the collection of complete information on the efficacy and safety of the medicine would be unethical. These medicines are subject to specific post-authorisation obligations and monitoring.

Medicines for rare diseases

The EU framework for orphan medicines encourages the development and marketing of medicines for patients with rare diseases by providing incentives for developers.

Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity. Among the 104 medicines recommended for marketing authorisation in 2025, 16 had their orphan designation confirmed by the end of the year.

Five medicines lost their orphan status before receiving a marketing authorisation, which means they were still authorised as medicinal products but not as orphan medicinal products. These are: Attroggy, Aucatzyl, Blenrep, Dawnzera and Oczyesa.

New uses for existing medicines

In 2025, 89 extensions of indication were recommended, including 40 for paediatric use. The extension of the use of a medicine that is already authorised for marketing in the EU can also offer new treatment opportunities for patients. Notable extensions of indication included:

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  Fabhalta, for the treatment of adults with complement 3 glomerulopathy, an ultra-rare kidney disease that previously had no treatment options. Fabhalta was initially approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria who have haemolytic anaemia.

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  Ixchiq, for active immunisation of adolescents from 12 years of age against the disease caused by Chikungunya virus. This vaccine was initially approved to protect adults against the disease. Chikungunya is transmitted to humans by infected mosquitoes.

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  Kaftrio and Kalydeco, two cystic fibrosis medicines to be used in combination in patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This extended use for additional, rare mutations allows the treatment of about 95 % of all cystic fibrosis patients. Cystic fibrosis is an inherited disease that has severe effects on the lungs, the digestive system and other organs.

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  Mounjaro, for the treatment of adolescents and children from 10 years of age with insufficiently controlled type 2 diabetes, together with diet and physical activity. Mounjaro was initially approved for use in adults only.

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  Uplizna, for the treatment of active immunoglobulin G4-related disease, a rare autoimmune disease for which there were no authorised medicines in the EU. Uplizna was initially approved for neuromyelitis optica spectrum disorders.

Negative opinions

The Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion for seven medicines in 2025: Atropine sulfate FGK, Blarcamesine Anavex, Elevidys, Jelrix, Kinselby, Nurzigma and Rezurock.

Keeping patients safe

Monitoring medicines after their authorisation – optimising safe and effective use

EMA and the EU Member States continuously monitor the quality, safety and the benefit-risk balance of authorised medicines when they are used in real life. This is to optimise how the medicine is used by patients to achieve its full benefit and to protect patients from avoidable side effects. Regulatory measures range from a change to the product information to the suspension or withdrawal of a medicine or the recall of a limited number of batches.

Important new advice issued in 2025 included:

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  Azithromycin, new recommendations on the way azithromycin is used, including the removal of certain indications. These recommendations aim to optimise the use of this common antibiotic and minimise the development of antimicrobial resistance.

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  Caspofungin, new warning against the use of polyacrylonitrile-based membranes during continuous renal replacement therapy in critically ill patients receiving caspofungin.

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  Clozapine, recommendation to ease risk minimisation measures based on new scientific evidence showing a markedly reduced risk of severe neutropenia over time; the frequencies of blood monitoring have been reduced, and absolute neutrophil count will now be the sole parameter required, replacing the previous need to also measure white blood cell count.

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  Crysvita (burosumab), new recommendations to monitor blood calcium levels and parathyroid hormones due to risk of severe hypercalcaemia, and to add related possible side effects to the product information.

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  Finasteride and dutasteride tablets, enhanced measures to minimise risk of suicidal thoughts alerting patients treated for androgenetic alopecia about the need to seek medical advice if they experience problems with sexual function (such as decreased sex drive or erectile dysfunction) known to contribute to mood alterations and suicidal ideation in some patients.

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  Injectable tranexamic acid, a reminder to healthcare professionals to only administer injectable tranexamic acid intravenously (into a vein) and not by any other route.

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  Ixchiq (live attenuated chikungunya vaccine), recommendation to only administer the vaccine when there is a significant risk of chikungunya infection and after careful consideration of the benefits and risks.

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  Mysimba (naltrexone/bupropion), new measures to minimise potential cardiovascular risks with long-term use and an obligation on the company to provide more information from an ongoing study on the medicine’s cardiovascular effects in patients treated for more than one year.

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  Oxbryta (voxelotor), recommendation to maintain the marketing authorisation suspended, as recent clinical trials showed more sudden pain episodes and deaths in patients taking Oxbryta. This follows interim measures taken by the committee in September 2024, when it temporarily suspended the medicine to review emerging safety data.

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  Oxycodone, new black box added to the existing warning in the patient leaflet stating that oxycodone is an opioid that can cause dependence and/or addiction. Dependence and addiction are important risks of oxycodone and remain of concern in the EU/EEA.

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  Remsima (infliximab), instructions to healthcare professionals to confirm patients do not have hereditary fructose intolerance before using a new intravenous formulation or contraindication of a new intravenous formulation in patients with hereditary fructose intolerance.

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  Semaglutide, update of the product information to include non-arteritic anterior ischemic optic neuropathy (NAION) as a very rare side effect and to stop treatment with semaglutide if NAION is confirmed.

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  Tegretol (carbamazepine), restriction on the use of Tegretol 100 mg/5 mL in neonates because the concentration of the excipient propylene glycol exceeds the recommended threshold.

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  Varilrix and Varivax (varicella (chickenpox) vaccines), update to the product information to further describe the severity of the risk of encephalitis. People who receive the vaccine should seek immediate medical attention if they develop signs of infection or inflammation of the brain.

More information and figures on human medicines are available in Chapter 2.

Veterinary medicines

New medicines to benefit animal health in Europe

In 2025, EMA recommended 30 veterinary medicines for marketing authorisation — the highest number of recommendations in a year for a second consecutive year. This suggests a sustained high interest in the development of veterinary medicines and the positive impact of the 2022 Veterinary Medicines Regulation in fostering innovation.

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  Epizootic haemorrhagic disease vaccine (recombinant protein) Laboratorios Syva S.A, for the active immunisation of cattle to reduce viraemia (presence of viruses in the blood) and fever caused by epizootic haemorrhagic disease virus serotype 8.

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  Innovax-ND-IBD-ILT, for the active immunisation of one-day-old chicks or 18-19 day-old embryonated chicken eggs to reduce mortality and clinical signs caused by Newcastle disease virus and to reduce mortality, clinical signs and lesions caused by avian infectious laryngotracheitis virus, Marek’s disease virus and infectious bursal disease virus.

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  Vaxxinact H5, for the following indications associated with highly pathogenic avian influenza serotype 5, including the circulating clade 2.3.4.4b:

  • active immunisation to prevent mortality, clinical signs and to reduce viral excretion in chickens and mulard ducks;
  • to reduce mortality, clinical signs and viral excretion in muscovy ducks and turkeys;
  • to reduce viral excretion in pekin ducks.
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  Vaxxitek HVT+IBD+H5, for:

  • active immunisation of one-day-old chicks or 18-day-old embryonated chicken eggs to reduce mortality, clinical signs and virus excretion due to infection with highly pathogenic avian influenza virus of the H5 subtype, including the circulating clade 2.3.4.4b;
  • active immunisation of one-day-old turkeys to reduce mortality, clinical signs and virus excretion due to infection with highly pathogenic avian influenza virus of the H5 subtype, including the circulating clade 2.3.4.4b.

Optimising the safe and effective use of veterinary medicines

EMA and EU Member States continuously monitor the efficacy, safety and quality of the veterinary medicines on the market in the EU. The aim is to optimise their use to achieve full benefit, and to protect animals and users from avoidable adverse effects.

Important new safety advice issued in 2025

The product information for 13 medicines was updated based on new safety data, to help animal owners and healthcare professionals to make informed decisions when using or prescribing a medicine. These included:

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  Bravecto chewable tablets, changes to the product information to include pruritus (itching) as potential side effects in dogs.

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  Bravecto spot-on solution for cats, amendment to the product information to include pruritus and ataxia (incoordination) as potential side effects.

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  Bravecto spot-on solution for dogs, changes to the product information to include diarrhoea and pruritus as potential side effects.

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  Divence IBR Marker Live, Divence Penta and Divence Tetra, amendment to the product information to include milk production decrease, reduced food intake and decreased activity observed in dairy cows as potential side effects.

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  Eluracat, changes to the product information to include anorexia (loss of appetite), behavioural disorder, dyspnoea (difficulty breathing), loss of consciousness, sedation, recumbency (lying down), muscle weakness and hiding as potential side effects in cats.

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  Felpreva, amendment to the product information to change the frequency of application site reaction (e.g. scratching, erythema (reddening), hair loss, inflammation) from very rare to rare.

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  Librela, changes to the product information to include diarrhoea, emesis (vomiting), joint pain, lameness and swelling in multiple joints (immune-mediated polyarthritis), weakness (paresis), loss of movement (paralysis) and convulsion (seizure) as potential side effects in dogs. In addition, special precautions should be taken when treating dogs with the following pre-existing conditions: low amounts of red blood cells (immune-mediated haemolytic anaemia), lameness and swelling in multiple joints (immune-mediated polyarthritis), low amounts of platelets (thrombocytes) (immune-mediated thrombocytopenia) or when treating dogs with preexisting convulsion (seizure) disorder.

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  Mhyosphere PCV ID, amendment to the product information to change the frequency of elevated temperature from common to very common.

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  Neptra, changes to the product information to include facial paralysis (loss of movement) as potential side effects in dogs.

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  Osurnia, amendment to the product information to include new special precautions: in very rare cases, eye disorders such as neurogenic keratoconjunctivitis sicca, keratoconjunctivitis sicca, corneal ulcer, blepharospasm, eye redness and ocular discharge have been reported in treated dogs and ataxia (incoordination), internal ear disorder (mainly head tilt), facial paralysis and nystagmus (involuntary eye movements) have been reported in very rare cases in post-authorisation experience.

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  Yurvac RHD, changes to the product information to include anorexia and intestinal stasis (inactivity) as potential side effects in rabbits.

Protecting consumers against medicine residues in food of animal origin

If a medicine is intended to be used in a food-producing animal, it needs to be safe for people to eat the food that comes from this animal. EMA recommends maximum residue limits (MRLs) that reflect the level of residues of a veterinary medicine in food derived from a treated animal that can be considered safe for human consumption. The MRL is established before a medicine can be authorised for food-producing animals in the EU and entered in the annex to Commission Regulation (EU) No 37/2010.

In 2025, positive opinions were adopted recommending the extension of MRLs for the following active substances:

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  Fluralaner, extension to salmonidae and other fin fish.

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  Lidocaine, modification of the use restrictions in porcine.

More information and figures on veterinary medicines are available in Chapter 2.