VX-770

Question

Hi there,

My daughter is currently 7 months old with G551D as one of her mutations.

I just wondered if the drug is approved this year in the 6yrs and older subset can it then be used in younger children with the dosage ammended accordingly? Or would they need to carry out a clinical trial in younger children first?

Thanks,

Jess

Answer

Dear Jess,
Thank you for your question.
KALYDECO (ivacaftor, VX-770) is a medicine in development for the treatment of people with cystic fibrosis and the G551D CFTR mutation by a pharmaceutical company called Vertex. Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to help CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways.
The drug is currently being considered via accelerated assessment by the regulatory authorities for approval, which is granted to new medicines of major public health interest and shortens the review time.
The regulatory submissions are supported by results of Phase 3 study in children between the ages of 6 and 11. Patients receiving the treatment on the study experienced rapid, significant and sustained improvements across a variety of disease measures, including lung function. The majority of adverse events associated with KALYDECO were mild to moderate in severity and non-serious.
Whilst patients with CF younger than 6 years with the G551D mutation may stand to benefit from KALYDECO, before these studies been carried out, the effects of the drug in children younger than 6 years are unknown and the drug could not be indicated for use in this age group until a clinical trial has been completed demonstrating the drug to be safe and effective.
As Vertex prepares for the potential launch of KALYDECO for people with the G551D mutation, the company is planning to begin additional studies of KALYDECO in children with CF as young as two years of age.

Best wishes
Katherine O’Neill
Prof Stuart Elborn
Belfast

References
Vertex Pharmaceuticals Incorporated Press release January 8, 2012 Vertex Announces Key 2012 Business Objectives as Company Prepares for Planned Global Launch of KALYDECO in Cystic Fibrosis

19.01.2012

The answer is edited by: Prof Stuart Elborn