The Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the triple combination medication deutivacaftor/tezacaftor/vanzacaftor (brand name Alyftrek) to treat cystic fibrosis (CF) in individuals six years of age and older who have particular mutations in the CFTR gene that have been demonstrated to respond to treatment in clinical trials. This includes the most prevalent mutation that causes cystic fibrosis, F508del.
is a genetic disorder brought on by a malfunctioning CFTR gene, which controls the movement of water and chloride into and out of the lungs and other organs. Lung infections and issues with food digestion can result from this accumulation of sticky mucus in the digestive tract and lungs
As a CFTR "modulator," deutivacaftor, tezacaftor, and vanzacaftor are intended to fix the faulty protein produced by the CFTR gene in individuals with cystic fibrosis. One tablet of deutivacaftor, tezacaftor, or vanzacaftor is taken daily. During treatment, grapefruit-containing foods and beverages should be avoided.
These findings were based on two randomised phase 3 studies with 480 participants who were 12 years of age and older (studies 121-102 and 121-103). Additional data from a phase 3 open label research (study 121-105, Cohort B1) supports these findings. Deutivacaftor/tezacaftor/vanzacaftor was found to be more effective at lowering sweat chloride levels and as effective at improving lung function as ivacaftor/tezacaftor/elexacaftor (Kaftrio), a triple combination therapy that is currently approved.
The most common side effects in the clinical trials were headache (15.8%) and diarrhoea (12.1%). For the full list of all side effects reported with this medicine, see Section 4 of the Patient Information Leaflet (PIL) or the Summary of Product Characteristics (SmPC) available on the MHRA website.
As with any medicine, the MHRA will keep the safety and effectiveness of deutivacaftor/tezacaftor/vanzacaftor under close review. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Learn more: Triple combination medicine deutivacaftor/tezacaftor/vanzacaftor approved for cystic fibrosis - GOV.UK
