Burger line Burger line Burger line
Logo Logo Logo
Burger line Burger line Burger line
Sign in
Sign in

Vertex secures FDA clearance for infants’ cystic fibrosis therapy

Vertex secures FDA clearance for infants’ cystic fibrosis therapy

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for KALYDECO (ivacaftor) to treat children with cystic fibrosis (CF) aged between one and four months.

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results.

It is based on data from a cohort in a Phase III, 24-week, open-label trial assessing the safety, pharmacokinetics and pharmacodynamics of ivacaftor in CF patients with an ivacaftor-responsive CFTR mutation who are under 24 months old.

This cohort showed a safety profile identical to that found in older children and adults.

KALYDECO previously secured approval for use in the US and European Union for treating CF in children aged four months and older.

The medicine is currently available for use in more than 30 countries.

Vertex global medicines development and medical affairs executive vice-president and chief medical officer Carmen Bozic stated: “Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants.”

CF is a rare, life-shortening genetic disease that is estimated to impact over 88,000 people across the globe.

The progressive multi-organ disease affects the patients’ lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.

May 4, 2023


0
Cart Subtotal:
Go to cart
You will be able to Pay Online or Request a Quote

We use "cookies*  to ensure the functionality of our website, recognise your browser or device, learn more about your interests, and provide you with essential features and services and for additional purposes, including:

Recognising you when you sign-in to use our services. This allows us to provide you with product recommendations, display personalised content, and provide other customised features and services.
Keeping track of your specified preferences. You may set your preferences through Your Account..
Keeping track of items stored in your shopping basket and personal cabinet.
Conducting research and diagnostics to improve ChemDiv’s content, products, and services.
Delivering content, including ads, relevant to your interests on ChemDiv’s site
Reporting. This allows us to measure and analyse the performance of our services.

By  cookies you give consent to the processing of your personal data, including transfer to third parties. Further information can be found in our privacy policy.

Accept all cookies