In 2025, the European Medicines Agency (EMA) recommended 104 new medicines for human use, almost 40% of which contained a completely new active substance. In a summary of 2025 achievements in medicines regulation, Emer Cooke, the EMA’s executive director, described 2025 as “a special year” for the agency, marking its 30th anniversary.

Regulatory decisions that she highlighted were the first treatment to delay the onset of stage 3 type-1 diabetes, the first treatment against Duchenne muscular dystrophy in patients from the age of 6 years who can walk, as well as lifting a restriction on the first vaccine in the EU to protect against Chikungunya.

The agency also recommended changes to how azithromycin is used following a review and re-evaluation. The aim was to optimize use and minimize the development of antimicrobial resistance, which had been increasing in prevalence in the European region. Changes included removing certain indications and a new product warning.

Baldness Drug Suicide Risk

Measures to minimize the risk for suicidal ideation with finasteride and dutasteride were also recommended in 2025. The agency said that the benefits of both drugs, used by men to treat androgenetic alopecia, continued to outweigh their risks, but urged healthcare professionals to alert patients to the risk and advise them to stop treatment and seek medical advice if they experience depressed mood, depression, or suicidal thoughts.

Diabetes: Landmark Regulatory Recognition

The EMA’s recommendation to grant marketing authorization for teplizumab (Tzield, Sanofi) to delay the onset of stage 3 type 1 diabetes in patients aged 8 years or older with stage 2 type 1 diabetes was “a landmark regulatory recognition that autoimmune diabetes has a modifiable preclinical phase,” said Chantal Mathieu, head of clinical endocrinology at KU Leuven, a research university in Leuven, Belgium. She told Medscape News Europe that the key question for future practice is “how early we can, and should, intervene.”

“To unlock the full potential of disease-modifying therapies, systematic population-level screening for islet autoantibodies will be essential to identify individuals at risk before irreversible beta-cell loss occurs,” she said. “This must be accompanied by greater regulatory flexibility to support immune-modulatory trials in children and adolescents.”

Mathieu, a former president of the European Association for the Study of Diabetes, said the development of such drugs was a major advance that signaled “a shift toward prevention-oriented, disease-modifying care in type 1 diabetes, provided screening, clinical trial design, and regulatory pathways evolve in parallel.”

Other key EMA drug approvals in 2025 included:

• The first oral drug to treat postnatal depression.
• An exceptional circumstances positive opinion for a new drug to treat maple syrup urine disease.
• A novel antibody-drug conjugate, representing a new mechanism of action, to treat relapsed or refractory multiple myeloma.
• A new prophylactic treatment for hereditary angioedema that can reduce attacks by up to 80%.
• A new immune treatment for refractory thrombocytopenia.
• A new estrogen endocrine agonist for locally advanced or metastatic breast cancer with an activating ESR1 mutation.
• Extension of the licensed indications for tirzepatide in type 2 diabetes to allow use in children and adolescents.
• An exceptional use recommendation for a drug to be used for acute radiation syndrome in nuclear emergencies.
• A combination agent orphan medicine to improve lung and digestive function in cystic fibrosis.

Modernizing Pharmaceutical Legislation

In December, the European Parliament and Council reached agreement on revising and modernizing the EU’s 20-year-old pharmaceutical legislation to boost innovation and investment in the EU’s pharmaceutical sector. The European Commission said that the new EU general pharmaceutical legislation rules will expand access to and availability of medicines, accelerate medicine supply chains, boost pharmaceutical innovation, enable timely market entry for generic medicines, and establish an EU framework to address medicine shortages, with a stronger coordination role for the EMA.

Changes will include cutting red tape for companies, reducing evaluation times for new medicines, and introducing regulatory sandboxes for secure testing of truly novel medicines, along with adapted frameworks for certain nonstandard treatments like personalized therapies. In addition, fulfillment of unmet medical needs will receive strong recognition. 

Agile Regulatory Framework

Commenting on the year’s decisions to Medscape News Europe, Nathalie Moll, director general of trade body for the European Federation of Pharmaceutical Industries and Associates, said, “We are pleased to see the EMA continue supporting the timely availability of innovative medicines for patients.”

“To maintain this momentum in the years ahead, it will be important for Europe’s regulatory framework to stay agile and driven by advances in science and technology,” she said.

Cooke said that the EMA’s efforts in 2026 will be focused on three areas: 

• Adapting the regulatory system under the recently strengthened legislative framework to deal with rapid scientific and technological advances, including increasing efficiency through smart digitalization and the use of artificial intelligence.
• Supporting innovation in public and animal health to enable new medicines to be authorized in the EU as rapidly as possible.
• Continuing to invest in the workforce of the European medicines regulatory network to help build capacity and capability for future challenges.