The US Food and Drug Administration (FDA) has granted Vertex Pharmaceuticals’ new drug application and its request for a six-month priority review of Kalydeco (ivacaftor), used to treat cystic fibrosis.

Kalydeco, as a cystic fibrosis transmembrane conductance regulator potentiator, helps to hydrate and clear mucus from the airways.

The approval is backed by results from two Phase III studies, STRIVE and ENVISION, in which cystic fibrosis patients treated with Kalydeco experienced rapid and sustained improvements across a variety of disease measures, including lung function.

The STRIVE study, which involved patients in patients aged 12 and older with at least one copy of the G551D mutation, reported a mean absolute improvement in lung function of 10.6% points.

A relative mean improvement in lung function of 16.8% from baseline through to week 24 was also observed among those treated with Kalydeco compared with the placebo.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The second pivotal Phase III study, ENVISION, evaluated the efficacy of Kalydeco in children with cystic fibrosis aged between six and 11 years, and had at least one copy of the G551D mutation.

Vertex global research and development chief scientific officer and executive vice-president Peter Mueller said that, if Kalydeco is approved by the FDA, it will be the first treatment to target the underlying cause of cystic fibrosis.

The company said that its marketing authorisation application for Kalydeco has been validated by the European Medicines Agency (EMA).

"The commitments by the FDA and the EMA to expedite their reviews of our applications underscore the significant potential of Kalydeco to help people living with cystic fibrosis," Mueller said.

Caption: Vertex receives FDA new drug application approval for Kalydeco.