"More than  years after the gene behind cystic fibrosis http_Genomeposterschromosomecftr.shtml was identified a pill that could fix the problem in some people is getting closer to reality. The experimental medicine taken twice a day goes by the code name VX. Now there are some promising results from a study httpclinicaltrials.govctshowNCTtermvxrank in  people with a specific genetic mutation that affects about four percent of cystic fibrosis patients. Lung function got  percentage points better on average in people who got the drug compared with those taking a placebo. The improvement at six months was still present almost a year after the studys start. That much improvement is a big deal for CF patients Robert Beall http president of the Cystic Fibrosis Foundation told Shots. It is larger than any other clinical intervention weve ever had in cystic fibrosis. Beall said there were no significant side effects. The foundation has championed the development of drugs including VX and has provided  million to Vertex http to get the medicine this far. If the drug makes it to market the foundation will receive royalties from sales. This pill for people with the GD mutation http of the cystic fibrosis gene helps a defective protein in cell membranes httpghr.nlm.nih.govgeneCFTR do a better job moving things around. Lisa Jarvis has more details httpcenblog.orgthehaystackvertexunveilsexcitingdataforcysticfibrosisdrug on that at the Haystack blog. But a key takeaway is that the medicine gets at a root cause of cystic fibrosis in some people rather than just relieving their symptoms. Article continues after this message from our sponsor Now to be perfectly clear all we know about the latest data is from press releases. And the drug hasnt been submitted for review by the Food and Drug Administration though Vertex expects to do that later this year. But these results even with plenty of caveats suggest the drug is on the right track. Vertexs shares jumped  percent Wednesday as investors figured VX looks even better than theyd expected. Wow wrote Dr. Mark Schoenebaum http a biotech and drug analyst ISI Group in a note to investors. The effectiveness of VX looks best case he wrote. Ultimately the drug might be used by around  patients worldwide if approved and Schoenebaum expects it would be command a very high price perhaps  a year."