EMA’s 2023 Highlights: Advancements in Human Medicines

20 January 2024

The European Medicines Agency (EMA) has concluded its annual review for 2023, providing a comprehensive overview of the authorization and regulation of new medicines. The year showcased significant achievements and advancements in various therapeutic areas, addressing unmet medical needs and global health challenges. In this report, we delve into the key figures, noteworthy medicines, regulatory procedures, and ongoing efforts to ensure the safety and effectiveness of pharmaceutical products.

In 2023, the EMA’s recommendations for the authorization of new medicines were marked by 77 positive opinions, 3 negative opinions, and 19 withdrawn applications. Among these, 39 new active substances, 3 PRIME-designated medicines, 17 orphan drugs, and 8 biosimilars were approved. A groundbreaking achievement was the approval of the first advanced therapy medicinal product utilizing CRISPR/Cas9 gene-editing technology to treat two rare blood disorders. Additionally, the year saw the approval of 14 generics, 3 accelerated assessments, 8 conditional marketing authorizations, and 1 approval under exceptional circumstances. These regulatory pathways reflect a strong commitment to facilitating early access to medicines and addressing urgent medical needs.

Concerning medicines recommended for use outside the European Union, in December 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted positive opinions for Arpraziquantel and Fexinidazole Winthrop under the EU-Medicines for All (EU-M4All) program. Arpraziquantel provides a new treatment option for schistosomiasis, while Fexinidazole Winthrop expands its use to treat the more acute and lethal form of human African trypanosomiasis. Amid the ongoing battle against the COVID-19 pandemic, EMA recommended Bimervax as a booster for individuals aged 16 and older who had previously received an mRNA COVID-19 vaccine. The approval of Bimervax was part of the ‘OPEN’ initiative, emphasizing international collaboration in the EU review of COVID-19 medicines. Furthermore, adaptations to existing vaccines were made to address the Omicron XBB.1.5 variant.

Three medicines received recommendations for marketing authorization following accelerated assessments, emphasizing the importance of optimizing data generation and enabling accelerated assessments for promising medicines targeting unmet medical needs. Eight medicines received conditional marketing authorizations, providing early access to new medicines addressing unmet medical needs. One medicine was authorized under exceptional circumstances, to grant access to medicines for rare diseases where comprehensive data may be challenging to obtain ethically or due to limited patient populations. In 2023, the EU framework for orphan medicines continued to support the development of medicines for rare diseases. Seventeen medicines had their orphan designation confirmed.

The CHMP adopted negative opinions for three medicines in 2023, emphasizing the agency’s commitment to maintaining a positive benefit-risk balance. Additionally, 77 extensions of indication were recommended, including 38 for pediatric use, providing new treatment opportunities for patients.

Ensuring Patient Safety beyond the authorization process

The agency continuously monitors the quality, safety, and benefit-risk balance of medicines in clinical practice. Regulatory measures, such as changes to product information, suspension or withdrawal of medicines, or batch recalls, are implemented to optimize the use of medicines and protect patients from avoidable side effects.

Integrity of Clinical Trial Conduct and Manufacturing

In a globalized pharmaceutical landscape, EMA also ensures adherence to EU standards irrespective of the location of clinical trials or manufacturing. The suspension of marketing authorizations for over 350 generic medicines tested by Contract Research Organisation (CRO) located in Pune (India), following a good clinical practice inspection, reflects the agency’s commitment to upholding rigorous standards in medicine development and manufacturing.

You can download the full report or read it online