Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has released the latest data from drug trials of its cystic fibrosis treatment, and investors are excited about those results. Today the company issued a statement saying that its two-drug treatment was again successful in treating cystic fibrosis, which means the company will likely receive approval from the Food and Drug Administration. Today’s announcement follows positive results from a previous drug trial, which were reported in April.
Vertex’s drugs work
The data shows that Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)’s drug pairing improved patients’ lung function while also cutting down on pulmonary flares. Vertex management told The New York Times that the results of the test at least met their optimistic expectations. CEO Dr. Jeffrey Leiden said they will be submitting applications for approval of the drug combination in both the U.S. and Europe during the fourth quarter of this year.
Nonetheless, the results weren’t quite as good as investors and doctors were hoping. The results show an improvement of between 2.6 and 4 percentage points compared to those who were given a placebo. This was toward the low end of what they had hoped. Some doctors had been hoping for 6% to 7%, while others said most patients wouldn’t even notice a 3% improvement in lung function initially. However, they also said that if the effects lasted more than six months, it would make a difference.
How Vertex’s drug pairing works
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) did receive FDA approval for Kalydeco, one of the two drugs in the study, in 2012. However, the drug only works for approximately 4% of patients—those who have a certain mutation. This recent drug trial combined Kalydeco with another drug developed by Vertex. The new drug that’s under testing is known as lumacaftor. By combining the two drugs, Vertex said almost half of those who suffer from cystic fibrosis would be helped. The treatment covers those who have two copies of the most common gene mutation that causes cystic fibrosis.
Previous treatments include antibiotics and an enzyme that loosens the mucus that causes permanent lung damage and repeated infections in those with cystic fibrosis. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)’s Kalydeco drug was the first one to actually impact the genetic defect that causes the disease. Vertex hopes its new drug lumafactor will be the second one.