La European Medicines Agency The European Medicines Agency (EMA) has taken a significant step by recommending the approval of a dozen new drugs and the expansion of use for several existing treatments. These decisions, taken at the February meeting of its Committee for Medicinal Products for Human Use (CHMP), affect a wide range of conditions, from rare diseases and childhood cancer even the flu and Covid-19.
Beyond the purely technical impact, the CHMP resolutions paint a picture in which both the therapeutic options in Europe such as support for countries with fewer resources. Among the medicines that have received the green light are a new combination mRNA vaccine, easy-to-administer oral treatments, and drugs targeting diseases that, until now, had limited alternatives.
New medicines: 12 proposed authorizations
At their last meeting, the EMA CHMP has proposed the authorization of 12 new drugsIn addition, it recommends expanding the indications for six other drugs already available in the European Union. The list includes cancer therapies, treatments for neurological and hormonal disorders, drugs for rare diseases, and several biosimilars that aim to increase competition and access.
Among these new features, a significant package of six biosimilars, entre ellos two insulins for diabetesa biosimilar of etanercept for inflammatory joint and skin diseases, another of pertuzumab For breast cancer, a biosimilar of tocilizumab Indicated in conditions ranging from rheumatoid arthritis to serious complications following therapies CAR-T, And a teriparatide intended for patients with osteoporosis. These alternatives aim to to promote greater sustainability of European healthcare systems.
Advances in pediatric oncology and rare diseases
Among innovative medicines, one that has attracted the most attention is Ojemda (tovorafenib), for which the CHMP proposes a conditional marketing authorizationIt is aimed at patients from six months of age with low-grade pediatric glioma, a brain tumor considered benign but which can seriously compromise the child's quality of life.
Until now, the approach to this type of glioma has relied primarily on the surgery and chemotherapyAlthough chemotherapy can be effective in some patients, its benefit is usually limited and is associated with Side effects important, especially delicate in young children. In this context, Ojemda presents itself as a weekly oral administration option, designed to cover a broader spectrum of low-grade pediatric glioma cases.
Another important development in the field of rare diseases is Xolremdi (mavorixafor), which has received a positive opinion from the CHMP in Exceptional circumstances for the treatment of WHIM syndromeThis is an extremely rare inherited condition in which the immune system does not function properly, predisposing individuals to recurrent bacterial and viral infections There is already an increased risk of some virus-associated cancers. The drug is indicated for patients 12 years of age and older.
In the field of rare endocrine diseasesThe committee has also given a favorable assessment to Palsonify (paltusotin) for the acromegalyThis rare hormonal disease usually appears in middle age and is caused by an excessive production of growth hormone by the pituitary gland, with consequences such as abnormal growth of hands, feet and facial features, as well as cardiovascular and metabolic problems.
New options for Parkinson's disease, chronic urticaria, and other conditions
The CHMP has issued a favorable opinion for Onerji (levodopa/carbidopa), a medicine intended to adults with advanced Parkinson's diseaseThis progressive neurodegenerative disease is characterized by tremors, muscle rigidity, slowness of movement, and balance problems, and often requires complex therapeutic adjustments as it progresses. The new formulation of Onerji seeks to improve the management of motor symptoms in advanced stages of the disease.
In the field of dermatological and immunological diseases, the committee has endorsed Rhapsid (remibrutinib) for the treatment of chronic spontaneous urticariaThis condition involves persistent outbreaks of hives and itching without a clear trigger. For those who do not respond adequately to conventional antihistamines, the availability of new options like Rhapside can represent a significant improvement in symptom management.
Along with these drugs, the EMA has also recommended the marketing of mCombriax, described as the first combined messenger RNA vaccine against seasonal flu and Covid-19. It is intended for people over 50 years of age, a group in which both infections tend to cause more severe illness and respiratory complications.
The agency notes that, despite the change in the pandemic's phase, Covid-19 continues to exert significant pressure on public health: The WHO has recorded more than 281 million cases in the European region until February 2026. In parallel, the seasonal flu continues to cause every year until 50 million symptomatic cases in the European Economic Area. The idea behind this combined vaccine is to simplify prevention, making it easier for at-risk individuals to protect themselves against both diseases with a single immunization.
Support for low-income countries: acoziborol for sleeping sickness
In addition to the decisions that directly affect the European market, the committee has issued a positive opinion for Acoziborole Winthrop (acoziborol), a single-dose oral treatment for Human African trypanosomiasis, popularly known as sleeping sickness, caused by the parasite Trypanosoma brucei gambienseThis medicinal product is intended for use outside the European Union and has been evaluated using the procedure EU-M4All.
The EU-M4All program allows the EMA to contribute to regulatory decisions of low and middle income countriesaccelerating access to essential medicines in resource-limited settings. In the case of Acoziborole Winthrop, the aim is to significantly simplify treatment in both the early and secondary stages of sleeping sickness, which until now required more complex treatment regimens and, sometimes, prolonged hospitalization.
La African trypanosomiasis (Gambian disease) It is transmitted by the bite of infected tsetse flies. In the first few days after infection, fever, headache, and skin rashes may appear, but if the infection progresses without treatment, it eventually affects the central nervous systemwith sleep disturbances, behavioral changes, and ultimately, risk of death. Although control campaigns have significantly reduced the number of cases—currently fewer than 1.000 are reported annually in Africa—the disease remains potentially fatal if left untreated.
Acoziborole Winthrop is indicated in adults and teenagers from 12 years old that weigh at least 40 kg, covering both the first and second stages of Trypanosoma brucei gambiense infection. The fact that it is administered in a single oral dose may facilitate its use in rural areas or areas with limited healthcare infrastructure, where monitoring longer treatments is particularly difficult.
Rejected medicines and withdrawal of an application
Not all the proposals considered by the committee were adopted. The CHMP has recommended deny marketing authorization de Daybu (trophinitide), a drug developed for the Rett syndromeThis genetic disorder is characterized by intellectual disability, language loss, and regression of skills between 6 and 18 months of age. According to the EMA, the available evidence has not demonstrated a favorable benefit-risk balance.
It has also received a negative review. Iloperidone Vanda Pharmaceuticals (iloperidone), which had been considered for the treatment of schizophrenia and for manic or mixed episodes associated with Bipolar disorderAgain, the committee concluded that the data provided were not sufficient to support its authorization in the European Union under the requested conditions.
In parallel, it has been reported that withdrawal of the initial authorization application de Zumrad (sasanlimab), a drug in development for the bladder cancerWithdrawal implies that the applicant has decided not to continue with the evaluation process at this time, presumably awaiting new data or a reassessment of the clinical development strategy.
Expansion of indications for already authorized medicines
In addition to the new drugs, the CHMP has proposed extend therapeutic indications de six medications which already had authorization in the European Union. Among them, one of the most notable changes is that of dupixent (dupilumab), which expands its use to the Chronic spontaneous urticaria in children aged 2 to 11 yearsWith this decision, he becomes the first available biological treatment for pediatric patients under 12 years of age with this disease.
The expansion of Dupixent represents a significant strengthening of the therapeutic arsenal in pediatrics, especially for families whose children do not respond to standard treatments. Chronic spontaneous urticaria in childhood can be very debilitating, and in the absence of targeted options, the margin for maneuver was quite limited until now.
Another relevant change occurs with Jorveza (budesonide), which extends its indication to the eosinophilic esophagitis in patients between 2 and 17 years old. This rare inflammatory condition of the esophagus can cause difficulty swallowing, chest pain, and feeding problems, especially in young children. Jorveza's specific formulation for children aims to facilitate administration and improve adherence to treatment.
Furthermore, the committee has recommended four additional indication extensions for known cancer drugs and immunomodulators: Keytruda, Olumiant, Scemblix y StellarAlthough the specific details of each expansion are not broken down in the statement, these decisions usually respond to new evidence that demonstrates its usefulness in other types of tumors, inflammatory diseases or subgroups of patients that were previously excluded from the technical data sheet.
Taken together, these extensions allow treatments previously limited to certain indications to be offered to a greater number of patients within the EU, taking advantage of the accumulated experience and the safety and efficacy data obtained after its use in clinical practice.
The CHMP's February decision package reflects a broad movement in which the EMA strengthens its regulatory role both in Europe and in resource-limited settings. The recommendation of 12 new drugs, the approval of a combined mRNA vaccine against influenza and Covid-19, the arrival of therapies for pediatric cancer and rare diseases, along with the expansion of indications for several key medications and the introduction of biosimilars, create a scenario in which treatment options are expanding for millions of patients, always under the premise of rigorously evaluating the balance between benefits and risks.
