Investing.com -- Shares in Vertex Pharmaceuticals Inc (NASDAQ:VRTX) surged more than 7% in after-hours trading after an FDA advisory committee voted 12 to 1 on Tuesday to recommend preliminary approval of its Cystic Fibrosis drug Orkambi.
If approved, Orkambi will be the first and only drug on the market to treat patients above the age of 12, who have two copies of the F508del mutation in the CFTR gene. Cystic fibrosis is caused by missing or defective proteins in a cell that result from mutations in the CFTR gene. Nationwide, there are roughly 8,500 people in the U.S. over the age of 12 with two copies of the mutation.
Orkambi combines two other Vertex drugs, Ivacaftor, which is also known as Kalydeco and Lumacaftor, a CFTR corrector. Lumacaftor is designed to correct the defect in the protein, while Ivacaftor can enhance its functioning potential, according to a Vertex statement.
"Today's positive recommendation brings the cystic fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people," Dr. Jeffrey Chodakewitz, Executive Vice President and Chief Medical Officer at Vertex said in a statement. "We look forward to continuing to work with the FDA and other regulatory agencies throughout the world to make Orkambi available to eligible patients as soon as possible."
The FDA is expected to make a final decision on approval of Orkambi by early-July.
Shares in Vertex Pharmaceuticals soared 9.41 points or 7.58% to 133.49 in after-hours trading.