KALYDECO found to improve lung function in cystic fibrosis patients

Patients aged six and over who suffer from cystic fibrosis could benefit from KALYDECO treatment, according to new data. Vertex Pharmaceuticals presented new information at the tenth Australian Cystic Fibrosis Conference last month (August), which showed that the twice-daily treatment of patients that have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene could lead to improvements in lung function.

KALYDECO is the first drug to treat the underlying cause of the disease when it is a result of a mutated or missing CFTR protein. The lack of this protein or a defection in it is caused by the CFTR gene being mutated. The drug helps the gene to function more normally, which can improve lung function and other symptoms of cystic fibrosis.

Global studies, STRIVE and ENVISION, were previously reported to have positive results with the drug when treating children over the age of six, adolescents and adults. Patients with at least one G551D mutation were given two 150mg doses a day and reported that their lung function improved and other disease symptoms abated slightly. The newest analysis of treatment, which was pooled across both studies, with KALYDECO was designed to assess the drugs safety and efficiency.

It was found that patients experienced improvements in lung function in the second week of the trials. This improvement was sustained throughout the entirety of the 48-week studies. The mean absolute lung function improvement measured at 11.2 percentage points through week 24 for the patients that received the drug, in comparison to those that were treated with a placebo. Through week 48 the mean improvement was measured at 10.9 percentage points. The safety level of KALYDECO was on par with the independent findings of both the trials.  

An ongoing extension study, entitled PERSIST, enrolled those that were involved with the previous studies so long as they met with certain criteria. It was found the improvement in lung function and other aspects of the disease were maintained for a total period of 96 weeks of treatment.