Regulation & Policy

China’s regulatory authority, the National Medical Products Administration, has approved Blenrep (belantamab mafodotin), a treatment for multiple myeloma which is a relatively rare blood cancer but with a global prevalence. In China, it is now the only B-cell maturation antigen (BMCA) targeting second-line therapy for the disease. “Blenrep provides a new and needed mechanism of action in therapy,” the developer GSK Plc said in a statement on 20 April. Blenrep is an antibody-drug conjugate consisting of a humanised antibody conjugated to a cytotoxic agent via a non-cleavable linker.

Marty Makary, commissioner of the US Food and Drug Administration, has invited developers of testosterone replacement therapies for men to approach the agency to discuss a possible augmentation for their products. Specifically, this means pursuing a potential new indication for low libido in men with idiopathic hypogonadism, or sexual disfunction without a known cause. “New and emerging data suggest there may be an opportunity to help men suffering from symptoms that significantly affect quality of life,” he said in a statement issued on 16 April.

A veterinary vaccine using RNA technology and originally developed by Merck & Co has received a positive opinion from the European Medicines Agency on the basis of multiple, positive studies in cats. The vaccine, Nobivac NXT HCPChFeLV, contains live attenuated strains of five feline viruses, one of which is a strain of the feline leukaemia virus. This virus strain suppresses the immune system and is a common cause of cancer in cats. It is species specific and does not affect humans.

A small peptide drug in development at Oculis Holding AG of Switzerland has received regulatory designations from agencies in both Europe and the US for its promise as a treatment for optic neuritis, a disease that can seriously impair vision. Called privosegtor, the candidate drug activates pathways in the brain in order to promote the survival of neurons after an inflammatory attack. Optic neuritis can be caused by autoimmune diseases such as multiple sclerosis, or viral infections such as mumps, measles or the flu, according to the American Academy of Ophthalmology. 

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.