Regulatory activity is underway to bring a new treatment for patients with thymidine kinase 2 deficiency (TK2d) to the European market, an ultra-rare disease that is characterised by progressive and severe muscle weakness. On 30 January, the European Medicines Agency issued a positive opinion for the treatment, Kygevvi (doxecitine and doxribtimine). It is expected to be reviewed by the European Commission in the second quarter for a marketing authorisation decision.
A non-invasive device using electrical fields to disrupt cancer cell division was approved by the US Food and Drug Administration on 12 February. Developed by Novocure Ltd, the therapy is designed to treat locally advanced pancreatic cancer and can be used in a home care setting. It is administered through electrically insulated adhesive patches that are placed on a patient’s skin and connected to an electric field generator.
After initially refusing to review a candidate mRNA vaccine developed by Moderna Inc for influenza, the US Food and Drug Administration has reversed itself and agreed to consider the product for a marketing authorisation. The decision was announced by Moderna on 18 February and follows a meeting between the agency and the company during which a compromise was reached.
Moderna Inc has asked for a meeting with the US Food and Drug Administration to query the agency’s decision to refuse to review its candidate mRNA vaccine for influenza. Effective 16 February, the agency hadn’t disclosed whether or not it would consider a follow-up discussion known as a Type-A meeting. This follows an event in early February when the director of the FDA’s biologics evaluation and research division at the agency, Vinayak Prasad, issued a refusal-to-file letter to Moderna overruling the advice of career staff at the agency.
The European Medicines Agency issued positive opinions for 104 new medicines in 2025 of which 38 were drugs with new active substances, according to the agency’s annual human medicines report issued on 15 January. At 38, the number is fewer than the 46 novel drugs recommended during the previous year, but no less impactful.
Citing Denmark as an example, the Trump Administration has issued revised guidance on childhood vaccination policy that lowers the number of immunisations recommended for all children from 2026 onward. The guidance was issued on 5 January and generated critiques from multiple sources who argued that the review lacked transparency. This was disputed by the Health and Human Services Secretary Robert F Kennedy Jr who said the changes followed “an exhaustive review of the evidence” and aligned US vaccine policy with that of other developed nations.
Novo Nordisk A/S has become the first developer of a weight loss drug to have its medicine approved by the US Food and Drug Administration as a pill. The decision, announced on 22 December, enables Wegovy (semaglutide) to be administered orally, in addition to its four earlier approvals as an injectable treatment. This puts the Danish company in a stronger position than before, relative to its competitor Eli Lilly and Co which only recently submitted an oral obesity treatment to the FDA for review.
BC Platforms AG of Switzerland is to collaborate with a US data company to significantly increase its access to patient information for real world evidence studies. The agreement with GeneVault Lifesciences of Cambridge, US, was announced on 18 December and is expected to give the Swiss company new data sources across Asia, the Middle East, Africa and Latin America. These regions represent some of the world’s most genetically diverse yet historically under-studied populations.
GSK Plc has received US approval for a new biologic to treat severe asthma, a disease driven high levels of white blood cells in the airwaves which can lead to inflammation and a shortness of breath. The Food and Drug Administration decision, announced on 16 December, will introduce a new drug to the market requiring dosing only twice per year. Exdensur (depemokimab) is a long-acting interleukin-5 (IL-5) antagonist. It prevents IL-5 from activating its receptor on eosinophils, thereby reducing an overproduction of the white blood cells.
Twelve former commissioners of the US Food and Drug Administration have rebuked proposed changes to vaccine policy at the agency saying that the measures will impede the development of new products to prevent infectious disease. Led by Robert Califf, the commissioners outlined their concerns in an article in the 3 December 2025 edition of The New England Journal of Medicine. Dr Califf was FDA commissioner until January 2025. Altogether, the group represents more than 35 years of FDA oversight and policymaking.