Pharmaceutical R&D pays off: 46 new innovative medicines to arrive in 2024
The European Medicines Agency approved a total of 114 medicines last year, 46 of which had a new active substance, seven more than the previous year.
Up to 15 of these new medicines are for the treatment of rare diseases.
Farmaindustria.es
Pharmaceutical companies’ continued investment in research and development of new medicines continues to pay off year after year. This is evidenced by the latest annual report on drug approvals from the European Medicines Agency (EMA), which shows that in 2024 the EMA recommended 114 medicines for marketing authorisation, of which 46 are novel therapeutic options as they contain a new active substance that has never before been authorised in the European Union. In addition, up to 15 of the new medicines are for diseases considered rare or infrequent.
Last year’s 46 new medicines are seven more than the 39 approved in 2023 and is in line with the average number of innovations approved each year, as the EMA has approved just over 200 new active substances in the last five years.
The European Medicines Agency itself highlights in its annual report Human Medicines in 2024 that among the new treatments approved ‘there are a number of medicines that stand out for their contribution to addressing public health needs or for the clear innovation they represent’. In the area of neurology, a new targeted medicine for the treatment of early Alzheimer’s disease and another for the treatment of adults with a type of amyotrophic lateral sclerosis (ALS); three medicines for rare diseases in the area of haematology: a gene therapy for the treatment of haemophilia B; a medicine for paroxysmal nocturnal haemoglobinuria; and the first monotherapy for haemolytic anaemia in patients with paroxysmal nocturnal haemoglobinuria. In addition, in the cardiovascular area, a medicine for pulmonary arterial hypertension, also considered a rare condition, was approved.
The report also highlights the approval of six medicines with the PRIME (Priority Medicines) designation, which means that they address unmet medical needs in diseases that have not yet been effectively treated. ‘The PRIME programme aims to help patients benefit as soon as possible from promising medicines that address an unmet medical need,’ says Isabel Pineros, head of Farmaindustria’s Pharmaceutical Provision and Access Department. ‘For this type of medicines, and for those with a clear therapeutic advantage, a special funding procedure is needed to allow patients to benefit from innovations as a matter of urgency, as outlined in Farmaindustria’s Proposal for Early Access to Innovative Medicines.
In addition, the EMA highlights in its report the approval of three other treatments for their innovation: the first needle-free and smaller form of adrenaline to treat allergic reactions, the first treatment for tumours associated with von Hippel-Lindau disease, and a new combination of antibiotics for the treatment of severe infections.
In addition, 90 indication extensions were recommended in 2024, of which 40 are extensions of medicines for paediatric use that are already authorised in adults, highlighting the commitment of pharmaceutical companies to include in therapeutic innovation and adapt medicines for paediatric patients.
In terms of therapeutic areas, oncology was the area with the highest number of new innovative medicines, with 13 approvals, followed by haematology, which had up to 10 new active ingredients, four of which were for rare blood diseases. The cardiovascular area also stood out, with the arrival of three medicines with new active ingredients.
It was also a good year for innovation in vaccines. The European Agency highlights the recommendation for approval of several new vaccines, including one to protect against Chikungunya disease and a new mRNA vaccine against lower respiratory tract disease caused by respiratory syncytial virus (RSV), as well as the expanded use of an mpox (monkeypox) vaccine to protect adolescents aged 12-17 years.
