US biotech company Vertex Pharmaceuticals (Nasdaq: VRTX) has received European Commission approval to extend the use of its cystic fibrosis therapy, Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with ivacaftor.
The updated authorization permits treatment for children aged two years and older who have at least one non-class I mutation in the CFTR gene. Previously, the therapy was approved for patients aged six and above with specific genetic mutations.
Cystic fibrosis is a genetic disease affecting more than 94,000 people in North America, Europe, and Australia, leading to progressive lung damage and other complications.
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2025 | Headless Content Management with Blaze