In a significant boost for global healthcare, the European Medicines Agency recommended 114 medicines for marketing authorisation in 2024, with 46 containing new active substances never before authorised in the European Union.
This represents a notable increase from the previous year, signaling a robust pipeline of innovative treatments reaching patients. These approvals span a remarkable range of medical challenges—from the first medicine to treat early Alzheimer's disease to novel cancer therapies and groundbreaking antibiotics. This article explores the most exciting developments from this year's approvals and what they mean for the future of medicine.
Medicines Recommended
New Active Substances
Oncology Products
Biosimilar Medicines
The EMA's regulatory activity in 2024 demonstrates a thriving pharmaceutical innovation ecosystem in Europe. While the U.S. Food and Drug Administration (FDA) approved 50 novel drugs in 2024 (down from 55 in 2023), the EMA increased its approvals of new active substances from 39 in 2023 to 46 in 2024 .
Beyond the headline figure of 114 recommended medicines, the 46 new active substances represent truly novel therapeutic approaches. Cancer remained the strongest therapeutic area, with 28 recommendations for oncology products alone. Additionally, the EMA recommended 28 biosimilar medicines, which help improve treatment accessibility and affordability for conditions ranging from cancer to osteoporosis and autoimmune diseases 1 .
This reversal of trends highlights Europe's growing attractiveness for pharmaceutical innovation and approval submissions.
The EMA recommended authorization of the first needle-free, compact adrenaline formulation for treating severe allergic reactions 1 . This innovation could dramatically improve emergency treatment for anaphylaxis.
In response to the growing threat of antimicrobial resistance, the EMA approved two new antibiotic medicines for severe infections, including Pfizer's Emblaveo (aztreonam-avibactam) for infections caused by aerobic Gram-negative bacteria resistant to other antibiotics 1 .
The agency recommended several innovative vaccines, including the first vaccine to protect adults against chikungunya virus and a new mRNA vaccine against respiratory syncytial virus (RSV)—demonstrating how mRNA technology pioneered for COVID-19 is now being applied to other infectious diseases 1 5 .
| Medicine | Therapeutic Area | Innovation |
|---|---|---|
| First Alzheimer's disease-modifying therapy | Neurology | First treatment proven to slow early Alzheimer's progression |
| Needle-free adrenaline | Allergy | First needle-free option for emergency anaphylaxis treatment |
| Marstacimab | Hematology | First approved anti-tissue factor pathway inhibitor for hemophilia |
| Belzutifan | Oncology | First treatment for von Hippel-Lindau disease-related tumors |
| Imetelstat | Oncology | First direct telomerase inhibitor for blood cancers |
Cancer treatment witnessed extraordinary advances in 2024, with oncology remaining the EMA's strongest therapeutic area. The shift toward precision oncology—treatments targeted to specific molecular features of tumors—continued to accelerate, with several tumor-agnostic approvals that treat cancers based on their genetic markers rather than their location in the body 2 .
The second quarter of 2024 alone saw 26 new or expanded indications for previously approved cancer drugs and 9 new oncology agents approved by the FDA and EMA. More than half of these approvals were for biologics or biosimilars, reflecting the industry's move toward large-molecule treatments 2 .
Therapies like trastuzumab deruxtecan (Enhertu®) became the first HER2-directed treatment to receive a tumor-agnostic indication, meaning it can treat various cancer types sharing the same molecular signature 2 .
The approval of multiple bispecific antibodies including epcoritamab, odronextamab, and amivantamab represents a new frontier in cancer immunotherapy. These sophisticated molecules can simultaneously bind to cancer cells and immune cells, effectively bringing them together to enhance tumor destruction 2 8 .
The EMA took important safety measures for CAR-T therapies, requiring warnings about the risk of secondary blood cancers, while also approving these innovative treatments for additional cancer types 2 .
| Therapy Type | Number of Approvals | Key Examples |
|---|---|---|
| Biologics | 20+ | Bispecific antibodies, monoclonal antibodies |
| Biosimilars | 3+ | Various cancer supportive care agents |
| Tumor-agnostic therapies | 4 | Entrectinib, repotrectinib, selpercatinib |
| CAR-T cell therapies | Multiple | Lisocabtagene maraleucel, idecabtagene vicleucel |
| PD-1/PD-L1 inhibitors | 9+ | Tislelizumab, dostarlimab, sugemalimab |
The remarkable therapies approved in 2024 rely on increasingly sophisticated research tools and methodologies. Here are some of the key technologies enabling these medical breakthroughs:
Complex protocols for genetically modifying patients' own T-cells to express chimeric antigen receptors that target specific cancer markers, revolutionizing blood cancer treatment 2 .
The success of COVID-19 vaccines accelerated adoption of mRNA platforms for other diseases, including RSV, with advantages in rapid development and manufacturing flexibility 1 .
Using detailed knowledge of protein structures to design precisely targeted small molecules, such as those inhibiting specific cancer-driving mutations 2 .
Developed alongside targeted therapies, these diagnostic tests identify patients most likely to benefit from specific treatments based on their genetic profile 8 .
Sophisticated biomarkers enable precise measurement of treatment effects and disease progression, particularly important in neurodegenerative diseases like Alzheimer's.
The approval of the first Alzheimer's disease-modifying treatment represents one of the most significant medical breakthroughs in recent decades. The development pathway offers fascinating insights into modern drug discovery.
The successful Alzheimer's therapy followed a rigorous clinical development program:
Decades of research established beta-amyloid as a key pathological feature of Alzheimer's, leading to the hypothesis that reducing these plaques could slow disease progression.
Researchers designed monoclonal antibodies specifically targeting beta-amyloid plaques while minimizing damage to healthy brain tissue.
Large-scale, randomized, double-blind trials enrolled patients with early Alzheimer's disease, using sophisticated biomarkers and cognitive assessments to measure treatment effects over 18-24 months.
The EMA's Committee for Medicinal Products for Human Use (CHMP) conducted a comprehensive scientific evaluation of the clinical data before recommending approval 3 .
Clinical trials demonstrated that treatment significantly slowed cognitive and functional decline in early Alzheimer's patients compared to placebo.
The therapy achieved its primary endpoint on established cognitive assessment scales, with treated patients showing approximately 30% less decline over the trial period. Additional biomarker data showed substantial reduction in amyloid plaque levels, confirming the drug's intended mechanism of action 1 .
This approval validates the amyloid hypothesis after previous failures and opens new possibilities for combination approaches that might deliver even greater benefits. It also demonstrates that neurodegenerative diseases, once considered untreatable, can be successfully targeted with modern therapeutic approaches.
| Aspect | European Medicines Agency | U.S. Food and Drug Administration |
|---|---|---|
| Novel drug approvals | 46 new active substances | 50 novel drugs |
| Orphan drug focus | 17 orphan-designated products | Over half of novel approvals were for rare diseases |
| Approval timelines | Standard 210-day assessment (proposed reduction to 180 days) | PDUFA deadlines with 89% met in 2023 |
| Notable unique approvals | Emcitate for Allan-Herndon-Dudley syndrome, Emblaveo antibiotic | Kisunla for Alzheimer's, Cobenfy for schizophrenia |
| Regulatory reforms | Proposed pharmaceutical legislation to streamline approvals | Food and Drug Omnibus Reform Act provisions |
The regulatory environment is also evolving, with proposed EU pharmaceutical legislation aiming to reduce standard assessment times from 210 to 180 days and introduce more flexible approaches like regulatory sandboxes for novel therapies 7 .
The EMA's 2024 recommendations represent more than just numbers—they signify hope for patients across Europe and beyond. From transforming fatal cancers into manageable conditions to addressing previously untreatable neurodegenerative diseases, these approvals mark significant milestones in medicine's relentless advance.
As regulatory processes evolve to keep pace with scientific innovation, and as researchers develop increasingly sophisticated therapeutic approaches, patients can look forward to more breakthroughs in the coming years. The 2024 harvest of new medicines demonstrates that even in challenging times, pharmaceutical innovation continues to deliver life-changing treatments for some of humanity's most devastating diseases.
The true impact of these approvals will unfold over the coming years as these medicines reach patients and their benefits are observed in real-world clinical practice. One thing is certain: 2024 will be remembered as a landmark year in which medical science overcame previously insurmountable therapeutic challenges.