Regulation & Policy

“The world is not waiting for us,” Ursula von der Leyen, the European Commission president told journalists on 29 January as she introduced new proposals to improve the competitive position of European industry. The Commission president outlined three goals for the coming year. They include providing incentives for new companies, especially in artificial intelligence; advancing clean energy; and expanding commercial partnerships with companies and countries across the globe. The measures for companies include incentives for generating risk capital. 

The Alzheimer’s disease drug, Leqembi (lecanemab), has been approved in the US for a new dosing schedule enabling patients to continue taking it after 18 months of treatment. The new maintenance indication will enable dosing once every four weeks using an intravenous administration. It will take effect after the 18-month initiation phase of bi-weekly dosing finishes. Leqembi has been approved for patients with mild cognitive impairment or mild dementia, also known as early Alzheimer’s disease.

A communications pause reportedly ordered by the Trump administration to the US federal health agencies and affecting their communications to the public and external partners had not been formally notified to the EU effective 24 January. The pause took effect on 21 January and is expected to continue through 1 February, according to the Washington Post, which obtained information about the pause from two internal memos. The pause was not publically announced. 

Citing the importance of the World Health Organization in maintaining global healthcare, a spokesperson for the European Commission said on 21 January that dialogue with the Trump Administration is the best way forward in addressing any shortcomings of the agency. “We need common solutions for common problems. We need to engage together,” she commented.

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.