A promising new medication for cystic fibrosis which is to be taken twice a
day has fared well in the mid-stage clinical trials. The research was published by Vertex in its press release on 23rd February 2011. Cystic fibrosis is a genetic disease
and can cause serious infections and digestive problems. It has been estimated
by the Cystic Fibrosis Foundation that about 30,000 US citizens while 8,500 UK
citizens are affected by the disease.
161 patients with a specific type of cystic fibrosis were part of the study.
They were asked to take this medication twice a day and it was seen that on an
average their lung function improved by 10% as compared to those patients who were
given placebo pills. This improved lung function is better than the other
treatments like inhaled medicines or enzyme replacement therapy for the
disease. Improvements in other areas like weight gain and reduction in
respiratory symptoms were also seen. Patients who took this medication showed
55% reduction in the probability of pulmonary exacerbation.
Vertex will submit this new medication for approval to the US FDA. The currently available drugs only target the symptoms of cystic
fibrosis while this new drug attempts to correct the malfunctioning protein in
the mutated gene's cell membrane that allows cystic fibrosis to flourish.