Drug for genetic disorder

Country

Netherlands

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.