A German company developing cell therapies to treat autoimmune diseases has been accepted into the European Medicines Agency’s support scheme PRIME, a regulatory pathway for small and medium-sized enterprises developing drugs for serious medical needs. The Heidelberg-based company, TolerogenixX GmbH, has received PRIME designation for its lead cell therapy candidate MIC-Lx for the treatment of immuno-suppression in live donor kidney transplantation.
The European Medicine Agency is updating its PRIME scheme, a programme of regulatory support for developers of novel medicines meeting a major public health need, in order to tighten reviews. The programme was introduced in 2016 to give special assistance to developers of advanced therapy medicinal products (ATMPs) and other new technologies largely produced by small and medium-sized enterprises.
The US Food and Drug Administration issued draft guidance on 18 March explaining how companies can move away from animal studies to alternative methods for testing new drugs. The agency first announced a plan to change the animal paradigm in April 2025.
Vinay Prasad, the chief medical and chief scientific officer at the US Food and Drug Administration and director of its biologics’ drug division, is to leave the FDA at the end of April. Dr Prasad’s departure was announced on 6 March by Marty Makary, the FDA Commissioner, and follows a period of disquiet within industry and patient advocacy groups about the way decisions have been made on new drug applications.
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.