Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 April 2025

EMA’s human medicines committee (CHMP) recommended 16 medicines for approval at its April 2025 meeting. The CHMP recommended granting a marketing authorisation for Alyftrek* (deutivacaftor / tezacaftor / vanzacaftor), a medicine intended for the treatment of cystic fibrosis in people aged six years and older who have at least one non class I mutation in the cystic fibrosis transmembrane conductance regulator gene. Cystic fibrosis is an inherited disease that damages the lungs, the digestive system and other organs. A positive opinion was adopted for Attrogy* (diflunisal), for the treatment of hereditary transthyretin-mediated amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves. The committee recommended granting a conditional marketing authorisation for Duvyzat* (givinostat), as a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who can walk. DMD is a rare, ultimately lethal genetic disease in which the muscles progressively weaken and lose function. See more details in the news announcement in the grid below. Sephience* (sepiapterin) received a positive opinion from the CHMP for the treatment of hyperphenylalaninaemia in adults and children with phenylketonuria, an inherited disease where people cannot process the amino acid phenylalanine, causing it to build up in the blood and brain, which can be harmful. Tepezza (teprotumumab) received a positive opinion from...