Regulation & Policy

A kinase inhibitor for the treatment of lung diseases has been recommended for use in children and adolescents from the age of 6 years by the European Medicines Agency. The treatment, Ofev (nintedanib), was authorised for adults in 2014 and subject to approval by the European Commission, will be available for children in 2025. The indication is progressive fibrosing interstitial lung disease which includes a group of rare respiratory disorders characterised by the build-up of scarring in the lungs which can lead to breathing difficulties.

A small molecule drug, Welireg (belzutifan), has been given conditional authorisation by the European Medicines Agency for tumours associated with von Hippel-Lindau disease and previously treated advanced kidney cancer. The opinion, announced on 13 December, expands the availability of the drug to Europe from the US where it was approved in 2021 by the Food and Drug Administration. The developer is Merck & Co Inc which submitted data to the EMA from two clinical studies showing efficacy.

A new cell therapy has been approved by the US Food and Drug Administration to treat paediatric patients from two months of age who experience complications from a stem cell transplant for blood cancer or an immune system disorder. The treatment, Ryoncil (remestemcell-L rknd), is an allogeneic therapy made up from mesenchymal stromal cells and isolated from the bone marrow of healthy adult donors. It is indicated for the treatment of acute graft-versus-host disease in patients undergoing transplants who do not respond to corticosteroids.

A treatment for classic congenital adrenal hyperplasia (CAH) has been approved by the US Food and Drug Administration – the first new drug in decades for a genetic disorder involving the adrenal glands. The drug, Crenessity (crinecerfont), is a small molecule to be used as an adjunct to glucocorticoids. In two randomised, placebo-controlled trials in adults and children Crenessity was able to reduce an overproduction of androgen in adults and children relative to the production of cortisol, an essential hormone. This was achieved without the need for high levels of glucocorticoids.

Autolus Therapeutics Plc, a UK-based cell therapy company, reached its first regulatory milestone on 8 November when the US Food and Drug Administration approved its chimeric antigen receptor (CAR) T cell therapy for cancer. The therapy, Aucatzyl (obecabtagene autoleucel), targets the CD19 antigen on B cells. The approval is to treat B cell acute lymphoblastic leukaemia (ALL), an aggressive type of blood cancer. It was based on evidence from a single-arm trial in adults whose disease had relapsed after two or more prior lines of therapy.

A new treatment for acute respiratory distress syndrome (ARDS) received a positive opinion from the European Medicines Agency on 15 November paving the way for its use in patients with a coronavirus infection. The treatment, Gohibic (vilobelimab), is a monoclonal antibody directed against a component of the complement system which is part of the body’s immune system. By targeting the complement component C5a, the antibody is believed to block an inflammatory response induced by severe SARS-CoV-2 infection. The treatment has been developed by InflaRx NV of Germany. 

A new gene therapy has been given an accelerated approval in the US for an inherited disorder affecting the way neurons transmit information to other cells in the body. The therapy, Kebilidi (eladocagene exuparvovec), has been shown to be effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare disease. It is caused by mutations in the DDC gene leading to shortages of the AADC enzyme which plays an important role in the nervous system.

After a second review, the main scientific committee of the European Medicines Agency has recommended approval of Leqembi (lecanemab) for the treatment of mild cognitive impairment due to early Alzheimer's disease in patients who have only one, or no copy, of the ApoE4 gene. The EMA opinion, announced on 14 November, will be forwarded to the European Commission for a decision. 

Mario Draghi, former president of the European Central Bank, has issued another appeal to policymakers to build an integrated capital market for the EU. In a commentary in the Financial Times on 2-3 November, Mr Draghi argues that a unified market would mobilise private capital to back projects in defence, the building of a digital infrastructure, and the mitigation of climate change. Currently the EU is home to more than 30 national and regional stock exchanges, each with its own regulations. A similar decentralised structure exists for bond markets.

A new oral medicine for schizophrenia was approved by the US Food and Drug Administration on 27 September introducing a mechanism of action for treating the disease – a leading cause of disability worldwide. The drug, Cobenfy (xanomeline and trospium chloride), is the first antipsychotic drug approved to treat schizophrenia that targets cholinergic receptors as opposed to dopamine receptors, which is currently the standard of care. The drug was developed by Bristol-Myers Squibb Co.