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As of December 31, 2024, a total of 112 drugs remained under evaluation by the European Medicines Agency (EMA), with 57 containing new active substances. The latest Horizon Scanning report from the Italian Medicines Agency (AIFA) provides a preview of therapies that could impact clinical practice in the coming months.

Each molecule awaiting approval represents both a therapeutic opportunity and a regulatory challenge, as many of the indications under review are in high-complexity clinical areas such as oncology, neurology, rare diseases, and advanced therapies.

Oncology Takes Center Stage

Among the 57 drugs with new active substances, oncology drugs continue to dominate, with 31.6% of the dossiers focused on this area, including both solid tumors and hematological diseases. One of the most anticipated candidates is linvoseltamab, a bispecific antibody targeting refractory multiple myeloma. If approved, it will be prescribed to patients who have failed at least three lines of treatment, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. 

Another promising drug is aumolertinib, for advanced non–small cell lung cancer with EGFR mutations. Lazertinib is also under evaluation for the same indication, likely to be used in combination with amivantamab, offering a more comprehensive approach to addressing molecular alterations.

Immunotherapy is also seeing significant progress. Tislelizumab, a checkpoint inhibitor already approved in other markets, is under review in Italy for new indications in colon and lung cancers. Lifileucel, a cell therapy based on tumor-infiltrating lymphocytes (TIL), could be used to treat nonresectable metastatic melanoma in patients who have failed other therapies. If approved, it would become the first TIL therapy available in Europe.

Gene therapy is advancing with the request for approval of nadofaragene firadenovec, which is expected to be the first gene therapy for high-risk, treatment-resistant bladder cancer.

Alzheimer’s, Amyloidosis, and Amyotrophic Lateral Sclerosis

In neurology, where advancements are often slow and controversial, 2025 appears to be a year of consolidation. Nine neurological drugs are currently under review, including two for Alzheimer’s disease. The most anticipated of these is blarcamesine, a candidate for treating early-stage Alzheimer’s disease. Clinical data presented so far show a 14% improvement in cognitive function and an 8% improvement in daily activities after 70 weeks of treatment. If approved, it could provide an alternative to lecanemab (recently approved for a subgroup of patients with Alzheimer’s disease) and donanemab (which was rejected in Europe in March 2025 due to a negative risk-benefit balance, with the company requesting re-examination). Blarcamesine may offer a different mechanism of action and potentially better tolerance.

Rare Diseases

A quarter of the new drugs under evaluation have received orphan drug designation, reflecting increasing interest from both researchers and the pharmaceutical industry in addressing unmet therapeutic needs. Among these drugs is delandistrogene moxeparvovec, the first gene therapy for Duchenne muscular dystrophy. This treatment, aimed at patients with specific mutations, promises to address the disease at its root, offering a more effective solution than the current symptomatic approach. Another notable therapy is obecabtagene autoleucel, a chimeric antigen receptor T-cell therapy for refractory or relapsed B-cell acute lymphoblastic leukemia. Both therapies fall under the category of “advanced therapies.”

In the field of rare cancers, there is growing interest in zanidatamab, a bispecific antibody for biliary tract carcinoma, which is a condition with poor prognosis and limited treatment options. Dorocubicel, designed for patients with hematologic disorders awaiting a transplant, also represents a potential lifesaving resource in critical situations.

Vaccines and Monoclonals

Prevention remains a key focus in 2025. Among the advanced dossiers is the monoclonal antibody clesrovimab, which appears effective in preventing respiratory syncytial virus (RSV) infection in neonates during the epidemic season. Its introduction could reshape vaccination strategies in pediatrics. New acellular pertussis vaccines aimed at providing perinatal protection through maternal immunization are also expected, along with mRNA-based RSV vaccines for adults older than 60 years. Finally, the chikungunya vaccine, which was already included in EMA’s PRIME scheme (an initiative launched by the EMA in 2016 to support the development of innovative medicines for unmet needs), was preapproved by the EMA in January 2025. It could receive final approval and become available in European vaccination centers.

Governance and Sustainability

As the number of drugs awaiting approval continues to rise, so does the pressure on the regulatory system. AIFA president Robert Nisticò commented, “The forecast for a record year in terms of new therapies entering the market is positive, but it presents the growing challenge of balancing accessibility with economic sustainability.” The high cost of advanced therapies, especially when potential users are limited, remains a primary concern.

In 2025, the new European Union (EU) Health Technology Assessment Regulation will begin to take effect. This regulation aims to standardize clinical evaluations across the EU, marking a key step in defining the therapeutic value of new technologies early on and positioning them within national healthcare systems.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.