In 2018, the European Medicines Agency (EMA) issued a total of 84 positive rulings on the authorisation of new medicines, of which 42 correspond to completely new drugs that include new molecules resulting from the effort of biomedical R&D, whose main driving force is the pharmaceutical industry. At a later stage, these rulings of the EMA become decisions for authorisation on behalf of the European Commission. The new drugs include three new gene and cell therapies, two of them based on CAR-T cells and intended for the treatment of certain blood cancers, and another for patients with a rare hereditary disease of the retina.

The different types of cancer, infectious diseases and neurological, metabolic and endocrine system pathologies made up the majority of the authorisations of new active ingredients, which increased by 20% compared to the previous year, when 35 completely new molecules were approved.

The medicines intended for the treatment of rare diseases also have an important presence, since 21 of the 84 positive rulings issued by the EMA during 2018 (one in four) correspond to these drugs. These include new drugs against alpha-mannosidosis, mucopolysaccharidosis type VII (Sly Syndrome), and myotonia.

In addition, several paediatric treatments have been approved, including those to treat West syndrome, insomnia in children and adolescents with autism spectrum disorders or with Smith-Magenis syndrome and neonatal diabetes.

The aforementioned paediatric medicines, the new gene and cell therapies and the above mentioned drugs against infrequent ailments are, according to the EMA, “significant advances in their respective therapeutic areas”.

Furthermore, over the past year four medicines were authorised following the accelerated procedure, allowing for the fulfilment of unmet medical needs. Of these, one is a new treatment to prevent episodes of haemorrhaging in certain variants of haemophilia A, two are to prevent against hereditary amyloidosis by transthyretin and the other is the first monoclonal antibody therapy to prevent attacks of hereditary angioedema.

On another note, the EMA gave its approval for 65 extensions for previously approved medicines, which include one related to a medicine against rheumatoid arthritis, which since this year can also be used to treat patients with Still’s disease, an unusual type of inflammatory arthritis.

Advances against cancer

As in the previous year, the largest group of new drugs that have received the green light from the European regulatory agency are aimed at addressing different types of cancer.

In total, there are 11 new oncological drugs, including the two aforementioned cell therapies and other treatments that represent new options for patients with cancers of the lung, prostate, breast, ovaries, fallopian tubes, peritoneum, melanoma and lymphoma.