Kalydeco: new treatment option for cystic fibrosis

Kalydeco (ivacaftor) is licensed for the treatment of cystic fibrosis in patients aged six years and over with a G551D mutation in the CFTR gene.

The recommended dose of Kalydeco is 150mg every 12 hours with fat-containing food.
The recommended dose of Kalydeco is 150mg every 12 hours with fat-containing food.


Ivacaftor is a selective potentiator of the CFTR protein. It increases CFTR channel gating to enhance chloride transport.1 


In two randomised, double-blind, phase III trials in 213 patients with the G551D mutation on at least one allele of the CFTR gene and forced expiratory volume in one second (FEV1) ≥40%, ivacaftor led to a rapid (15 days) improvement in FEV1. The treatment difference between ivacaftor and placebo for mean absolute change in per cent predicted FEV1 from baseline to week 24 was 10.6 percentage points in patients aged 12 years or older and 12.5 percentage points in patients aged 6 to 11 years.1


  1.  Kalydeco Summary of Product Characteristics, August 2012.

View Kalydeco record

Further information: Vertex Pharmaceuticals

Follow MIMS on Twitter 

Want news like this straight to your inbox?
Sign up for our bulletins

Have you registered with us yet?

Register now to enjoy more articles and free email bulletins

Already registered?
Sign in

More from MIMS

A woman wearing a white vest as a blue-gloved hand of a healthcare professional are seen injecting a vaccine.

'Nocebo' effect may cause two-thirds of COVID-19 vaccine reactions, study suggests

Two-thirds of adverse reactions reported with the COVID-19...

An elderly woman with one arm in a plaster cast sits resting both hands on her walker

Doctors urge NICE to reverse decision on new osteoporosis drug

More than NHS 100 clinicians have called on NICE to...

Drug shortages - live tracker

Use our constantly updated shortages tracker to check...

Image of both strengths of Seffalair Spiromax inhaler side by side.

New salmeterol/fluticasone dry powder inhaler for asthma

Seffalair Spiromax is the sixth dry powder salmeterol/fluticasone...