With the beginning of 2025, both the EMA and the FDA have released their annual reports on new drug approvals for the previous year. This post provides a comparative analysis of the approvals granted by both regulatory agencies, examining key areas such as orphan drug designations, special evaluation pathways, and differences in therapeutic areas.
With the beginning of 2025, both the EMA and the FDA have released their annual reports on new drug approvals for the previous year. This post provides a comparative analysis of the approvals granted by both regulatory agencies, examining key areas such as orphan drug designations, special evaluation pathways, and differences in therapeutic areas.
As 2025 begins, both the EMA and the FDA, the two most important drug regulatory agencies in the world, have recently published their annual reports on new drugs authorized throughout the previous year.
For the FDA, the report is published by the Center for Drug Evaluation and Research (CDER), which includes drugs based on chemical molecules and antibodies. Other products, such as vaccines, gene therapies, and cell therapies, are not included in this report, as their authorization depends directly on the Center for Biologics Evaluation and Research (CBER). Therefore, information about them is available on the CBER website. Each of these two FDA divisions is directly responsible for evaluating their respective drugs, although collaborations between them occasionally occur. For the following analysis, approvals from both divisions have been considered.
For the EMA, all types of therapies are included in its report, as all evaluating subdivisions fall under the CHMP.
The reports primarily highlight data on new innovative drugs (what the FDA refers to as "novel drugs" and the EMA as "new active substances"). Within this category, details are provided on which drugs target rare diseases, which have undergone special evaluation pathways, the therapeutic areas in which they are classified, as well as mentioning new biosimilars and new indications for already approved drugs in each region.
As a key statistic, during 2024, the U.S. approved 60 new drugs (50 from the CDER report + 10 from CBER), while Europe authorized 46.
Of all these drugs, only 14 appear on both lists and were authorized in 2024 by both agencies.
In fact, the CDER highlights in its report that 34 of the 50 new active substances (68%) were first approved in the U.S. before anywhere else in the world.
The trend of recent years continues, with oncology, hematology, and oncohematology being the areas with the highest number of new active substances. Some important drugs in this field include Beqvez, a gene therapy for hemophilia B, and Voydeya for residual hemolytic anemia.
It is also noteworthy that the FDA granted numerous approvals in neurology during 2024, including Kinsula, targeting Alzheimer’s disease, or Miplyffa, aimed at Niemann-Pick disease type C, both available in the U.S. but not yet in Europe, among others.
A significant difference exists in orphan drug approvals. In 2024, 26 (52%) of the total drugs approved by the CDER were designated as orphan drugs. In contrast, only 15 of the drugs authorized by the EMA were of this type (33%). Historically, the FDA has granted a significantly higher number of orphan drug designations and approvals than the EMA due to differences in regulatory requirements.
The following graph compares new orphan drugs reported by both agencies over the past five years.
Both agencies have different early access evaluation pathways designed to facilitate the approval of priority drugs for patients. Since the creation of these pathways, an increasing number of drugs have been designated under them.
In Europe, the special pathways include PRIME, accelerated assessments, conditional marketing approvals, and approval under exceptional circumstances. In the U.S., the corresponding pathways are fast track, breakthrough therapy, priority review, and accelerated approval. A single drug may follow more than one of these pathways depending on its characteristics.
The comparison between regulatory agencies is as follows:
Regarding new indications for already authorized drugs, the CDER report presents some examples but does not provide a total count. The EMA report is more precise, indicating that in 2024, 90 extensions of use were made, 40 of which targeted the pediatric population.
For biosimilars, the CDER highlights in its document that it set a record in 2024, with 18 biosimilars approved for 8 reference products. However, this figure is still below the 28 biosimilars recommended for authorization by the CHMP.
For more information, you can consult the full reports here:
