The recently published EMA document “Human Medicines Highlights 2023” (1) has shown that 2023 was a busy year for drug regulatory approval within Europe, with a total of 77 positive opinions given of the submissions received.
The largest area to receive drug approvals reported in this document is that of cancer (25 recommendation for approval), with many of these products containing new active substances as well as biosimilars and drugs with designated orphan status. A number of these new cancer treatments are noted in the document as representing “significant progress” in disease treatment namely Columvi (glofitamab), Tepkinley (epcoritamab), Elrexfio (elranatamab), Finlee (dabrafenib), Jaypirca (pirtobrutinib), Krasati (adagrasib), Lytogobi (futibatanib), Omjjara (momelotinib), Pedmarqsi (sodium thiosulphate) and Talvey (talquetamab).
The relatively high number of drugs receiving positive opinions from the EMA that are cancer treatments highlights where the main thrust of drug development is currently focused. Perhaps this is not surprising when one considers that the European Commission’s Joint Research Centre European Cancer Information System predicts a 21% increase in new cancer cases by 2040 compared to 2020 figures, with cancer-related deaths expected to rise by 32% during the same period (2). Both of these values are considerably above the predicted population growth rate for the same timeframe (2).
Eleven other areas where drug approval has been given are detailed in the document. These cover cardiovascular, dermatology, endocrinology, gastroenterology/hepatology, haematology/haemostaseology, Immunology/Rheumatology/transplantation, infections, metabolism, neurology, ophthalmology and pneumology/allergology. Three vaccines and three products as diagnostic agents were also recommended for approval.
Again, several areas have drugs that have been identified as providing significant progress. These are Aqumeldi (enalapril maleate) for the treatment of heart failure in children, Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy, Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent beta-thalassemia and sickle cell disease, Loargys (pegzilarginase) for the treatment of hyperarginaemia and Skyclarys (omaveloxalone) for the treatment of Friedreich’s ataxia.
Two vaccines also get special mention, namely Abrysvo for protection against respiratory syncytial virus (RSV) in infants and the over 60s and Arexvy also for protection against RSV in the over 60s.
Both of the vaccines mentioned above were approved via the accelerated assessment pathway, used to fast track approvals due to an unmet medical need.
The EMA has made good use of the various drug approval pathways available to it with several products in different medical areas being given accelerated assessments in one form or another including the use of conditional approvals for seven cancer treatments and one product, Loargys, gaining approval under exceptional circumstances. Rare diseases are also well presented in the EMA approvals with 17 medicines having their orphan status confirmed by the end of 2023.
As well as providing recommendations for drug licensing within Europe, the EMA also works with the World Health Organization and relevant national regulatory authorities to evaluate medicines for use in countries outside Europe which are experiencing difficult economic situations. In these countries internal drug regulation is more challenging. Medicines that fall into this category (EU-Medicines for all) are targeted to conditions that give the widest benefit to public health in that nation. In 2023 two medicines were assessed under this “EU-Medicines for all” initiative. These were Arpraziquantel and Fexinidazole for the treatment of schistosomiasis and trypanosomiasis respectively.
The EMA also issued 77 “extension of indication” for drugs, giving greater patient ranges for these products. Within this are included 38 products that are now deemed acceptable for pediatric use.
In terms of a comparison to 2022 (3), the number of positive opinions decreased from 89 to 77 in 2023 . However, the numbers for biosimilars and new active substances remained similar, with 8 biosimilars and 39 new active substances approved in 2023, compared to 8 biosimilars and 41 new active substances in 2022. The number of negative opinions remained unchanged at 3 for both years. Cancer treatment approvals were also consistent between the two years, with 25 approvals in 2023 and 23 in 2022 .
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