EMA’s Expected Drugs by 2025: Alzheimer’s to Cancer
new Medicines, Vaccines Await Approval in Italy
Table of Contents
- new Medicines, Vaccines Await Approval in Italy
- New Therapies Offer Hope for Rare Diseases, But Cost Concerns Loom
- Developing Story: Health updates
- New Medicines and Vaccines in Italy: A Q&A Guide
ROME – Italy’s regulatory agency, teh AMA, is currently evaluating 112 new medicines, with decisions expected by year’s end, according to the Horizon Scanning 2025 report recently published. The review includes 57 new active substances, 27 of which are designated as orphan drugs for rare diseases.
the potential approvals also include new vaccines targeting pertussis, respiratory syncytial virus (RSV), and chikungunya.
Oncology and Neurology Lead New drug Candidates
Of the new active substances under evaluation, a notable portion are for oncological and oncohematological conditions (31.6%), with 13 targeting solid tumors and five for blood cancers. The neurological area accounts for 15.8% of the drugs, including two for duchenne muscular dystrophy and one for Alzheimer’s disease.
Other therapeutic areas with multiple medicines under evaluation include metabolic diseases, infectious diseases, and hematology, each with 8.8% of the total.
Among advanced therapies with orphan drug designation, a treatment for Epidermolysis Bullosa has already received a favorable opinion from the AMA. Other orphan drugs under review target Fanconi anemia, Duchenne muscular dystrophy, acute lymphoblastic leukemia, hematological neoplasms requiring stem cell transplantation, and Wiskott-Aldrich syndrome. Non-orphan drugs are being considered for knee cartilage defects, melanoma, and bladder cancer.
Among non-orphan drugs, oncology and oncohematology treatments represent the largest group (30%), followed by infectious disease treatments, including vaccines (16.7%), and neurological treatments (13.3%). Hematology and psychiatry follow, each with 6.7% of the medicines under evaluation.
vaccine Pipeline
The expected new entries include several vaccines and preventative products for infectious diseases. A single-administration monoclonal antibody against respiratory syncytial virus (RSV), nirsevimab, is among those being considered.An acellular pertussis vaccine is also anticipated this year.
A 21-valent pneumococcal polysaccharide conjugate vaccine, already approved by the European Medicines Agency (EMA) in January, is aimed at adults aged 18 and over. This vaccine targets serotypes responsible for a majority of invasive pneumococcal disease cases and severe pneumonia,which,according to estimates from the Higher Institute of Health,lead to approximately 150,000 hospitalizations annually.
Alzheimer’s Drug Developments
Progress on new Alzheimer’s drugs has seen mixed results. The EMA deemed that the benefits of donanemab, intended for early-stage Alzheimer’s, “were not large enough to overcome the risk of possibly fatal events due to amyloid imaging anomalies.”
However, the European Commission confirmed market authorization for lecanemab, another new-generation drug for early Alzheimer’s, following a favorable opinion from the EMA in November. the outcome of the AMA’s evaluation of blarcamesine, a drug that experimental studies suggest improves cognitive skills, remains pending.
Targeting Tumors
In the oncohematological area, lindeseltamab is expected to become available following a recent favorable opinion from the AMA for treating adults with relapsed or refractory multiple myeloma. The advice is specific to patients who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, and have shown disease progression on the last therapy.
New drugs are also anticipated for melanoma, lung cancer, and breast cancer. These include mucoroselinib, a monotherapy drug for non-small cell lung cancer, and vorasidenib (with orphan designation) for treating astrocytoma and oligodendroglioma, cancers of the central nervous system with limited therapeutic options.
Psychiatric Advances
In psychiatry, the anticipated arrival of zuranolone offers a potential treatment for anxiety associated with postpartum depression.
New Therapies Offer Hope for Rare Diseases, But Cost Concerns Loom
European regulators are poised to approve a wave of innovative therapies, offering new hope for patients with rare and life-threatening conditions. Though, the anticipated influx of high-cost treatments is raising concerns about the sustainability of healthcare budgets.
Breakthrough Treatments on the Horizon
Among the promising new therapies is Obecabtagene Autoleucel, a treatment for acute lymphoblastic leukemia with refractory or recurrent B-cells. This therapy offers a potential lifeline for patients who have weary other treatment options.
For Duchenne muscular dystrophy, a genetic therapy called Delandistrogene Moxeparvovec is set to become available. This marks a significant advancement, as existing treatments primarily focus on managing symptoms. The new therapy targets a specific mutation, offering improved outcomes for a subset of patients, though it is indeed not a worldwide cure.
Expanded Indications and Biosimilars
Along with novel drugs, regulators are considering expanding the approved uses of 53 existing medications, encompassing 61 new therapeutic indications. Oncology and oncohematology lead the way, accounting for 42.6% of the potential new options, with 14 targeting solid tumors and 12 for blood cancers.
Other areas of focus include infectious diseases and vaccines (19.7%), hematology, gastroenterology, immunology-renovation, metabolism, and neurology (4.9% each), followed by endocrinology, pneumology, and psychiatry (3.3% each), and dermatology and nephrology-urology (1.6% each).
The expected approval of 35 biosimilar medicines, notably those related to the musculoskeletal system, and 10 generic medicines, is anticipated to generate cost savings.
Priority Medicines Program
The European Medicines Agency’s (EMA) PRIME (priority medicines) program highlights drugs with significant public health interest and therapeutic innovation for patients with unmet needs.as of January 2025, the program included 143 designations, with advanced therapies representing 45.4% of the total.
Oncology is the most represented area, with 27.3% of designations, followed by endocrinology-grain-fertility-metabolism (13.3%), hematology (12.3%), neurology (8.4%), vaccines (6.7%), infectious diseases (6.3%), ophthalmology (4.9%), and immunology-re-transplantation (4.2%).
By 2025, the EMA is expected to issue opinions on six of these medicines. Two have already been reviewed: a vaccine for the Chikungunya virus and Beremagene Geperpavec, a topical therapy for dystrophic epidermolysis bullosa. Other medicines under consideration include deoxymidine/dexytimidine for shy chinasi 2 deficiency, Mozafancogene Autothemcel for Fanconi anemia type A, Obecabtagene Autoleucel for acute lymphoblastic leukemia with refractory or recurrent B-cells, and Dorocubicel for urgent hematopoietic stem cell transplantation.
Cost Concerns and Sustainability
AIFA President Robert Nisticò acknowledged the promise of new therapies but cautioned about the financial implications. “the prediction of a new record year for the entry of new therapies on the market is good news, which, however, places us more and more in the face of the problem of how to guarantee the right balance between accessibility to treatments and economic sustainability,” he said.
Nisticò emphasized the need for a new approach to pharmaceutical expenditure, considering the broader economic benefits beyond healthcare.He noted AIFA’s efforts to renegotiate prices of older medications and expedite the introduction of generics. He also advocated for advanced health technology assessment (HTA) techniques to evaluate the cost-benefit ratio of new treatments,potentially including “bonus-malus” pricing based on real-world effectiveness data.
AIFA technical director Pierluigi Russo highlighted the importance of early identification and evaluation of new medicines and therapeutic indications. He noted that pharmaceutical expenditure is influenced by both innovation costs and social factors like an aging population, requiring collaboration among AIFA, regions, and national institutions to ensure lasting access to innovative treatments.
Developing Story: Health updates
This is a developing news story. Further details will be added as they become available.
check back for updates.
We are working to gather more facts and will provide a extensive report as soon as possible.
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New Medicines and Vaccines in Italy: A Q&A Guide
Welcome to a comprehensive look at the latest developments in new medicines and vaccines awaiting approval in italy. We’ll delve into the key areas, potential treatments, and the broader implications for healthcare. This Q&A style aims to provide you with a clear and informative overview.
General Background
What is happening with new medicine and vaccine approvals in Italy?
Italy’s regulatory agency, the Agenzia Italiana del Farmaco (AIFA), is currently evaluating a range of new medicines and vaccines.According to the Horizon Scanning 2025 report, AIFA is reviewing 112 new medicines, with decisions expected by the end of the year. This includes a important number of new active substances and vaccines, offering potential new treatment options for various health conditions.
What is the Horizon Scanning 2025 report?
The Horizon Scanning 2025 report,published by AIFA,outlines the upcoming developments in pharmaceuticals and provides a forward-looking view of the medicines and therapies that are expected to be approved and available in Italy.
Key Areas of Focus
What types of diseases are thes new medicines targeting?
The new medicines under evaluation cover a broad spectrum of therapeutic areas. Oncology and oncohematology lead with a significant portion of the new drugs, with many targeting solid tumors and blood cancers. Neurology receives considerable attention, including treatments for Duchenne muscular dystrophy and Alzheimer’s disease. other areas with multiple medicines under evaluation include metabolic diseases, infectious diseases (including vaccines), and hematology.
Are there any new vaccines expected?
Yes, several new vaccines are in the pipeline. The anticipated vaccines include:
Pertussis (whooping cough) vaccine: an acellular pertussis vaccine is expected to be available this year.
respiratory Syncytial Virus (RSV) vaccine: A single-management monoclonal antibody against RSV, nirsevimab, is also awaiting approval.
Pneumococcal Vaccine: A 21-valent pneumococcal polysaccharide conjugate vaccine, that targets serotypes responsible for a majority of invasive pneumococcal disease cases and severe pneumonia.
Chikungunya virus vaccines: Currently being reviewed as per the European Medicines Agency (EMA).
what are orphan drugs, and how many of those are being reviewed?
Orphan drugs are medicines developed for rare diseases, which affect a small number of peopel. Of the new medicines under review, 27 are designated as orphan drugs.
What are some specific examples of orphan drugs under review?
Examples of orphan drugs under review include treatments for:
Epidermolysis bullosa
Fanconi anemia
Duchenne muscular dystrophy
Acute lymphoblastic leukemia
Hematological neoplasms requiring stem cell transplantation
Wiskott-Aldrich syndrome
What role does the EMA play in this process?
The European Medicines Agency (EMA) plays a crucial part in the approval process. It evaluates medicines and provides opinions on their safety and efficacy. AIFA then uses these opinions to make its final decisions for the Italian market.
Specific Drugs of Interest
What’s the status of Alzheimer’s disease treatments?
The situation with Alzheimer’s drugs is mixed. The EMA deemed that donanemab, intended for early-stage Alzheimer’s, did not demonstrate significant enough benefits to overcome the risks. Though,the European Commission confirmed market authorization for lecanemab,another new-generation drug for early Alzheimer’s,following a positive EMA opinion. AIFA’s decision on blarcamesine, a drug suggested to improve cognitive function, remains pending.
What are the major oncology drug developments?
Multiple Myeloma: Lindeseltamab is expected to become available for adults with relapsed or refractory multiple myeloma.
Melanoma: New treatments are anticipated.
Lung Cancer: Mucoroselinib, a monotherapy for non-small cell lung cancer, is under review.
Central Nervous System Cancers: Vorasidenib (with orphan drug designation) is being considered for astrocytoma and oligodendroglioma.
Breast Cancer: Promising new treatments are also in the pipeline.
Are there new psychiatric medicines being considered?
Yes. zuranolone is anticipated, and it may offer a treatment option for anxiety associated with postpartum depression.
Broader Implications and Sustainability
What are the concerns about the cost of these new therapies?
What are the main concerns about new therapies?
The main concern is the financial impact on healthcare budgets. The influx of high-cost, innovative treatments poses a challenge in ensuring accessibility while maintaining economic sustainability.
AIFA President Robert Nisticò has highlighted this issue, emphasizing the need to balance treatment accessibility with financial responsibility.
What steps is AIFA taking to address these concerns?
AIFA is implementing strategies to manage pharmaceutical expenditure, including:
Renegotiating the prices of older medications.
Expediting the introduction of generic drugs.
Utilizing advanced health technology assessment (HTA) techniques to assess the cost-benefit ratio of new treatments, possibly, utilizing “bonus-malus” pricing based on real-world effectiveness data.
* Implementing priority medicines programs.
What is the European Medicines Agency’s (EMA) PRIME program?
The EMA’s PRIME (priority medicines) program supports medicines with significant public health interest and therapeutic innovation for patients whose needs are unmet. Oncology, endocrinology, metabolism, hematology, and neurology are amongst the most represented areas.
How is AIFA working with other institutions?
AIFA is collaborating with regions and national institutions to ensure lasting access to innovative treatments, recognizing that pharmaceutical expenditure is influenced by factors like innovation costs and the aging population.
Conclusion
The Italian regulatory landscape is poised for significant changes in healthcare with the anticipated approvals of new medicines and vaccines. While offering hope for patients, the focus is also on balancing access to these innovations with the long-term sustainability of healthcare systems. The next steps will be crucial in this regard. Keep checking back for further data as the story develops!