Vertex Pharmaceuticals Incorporated (VRTX) Receives FDA Approval for Cystic Fibrosis Treatment

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that the FDA has approved KALYDECO® (ivacaftor) for use in more than 600 people with cystic fibrosis (CF) ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This approval was based on Phase 3 clinical data for KALYDECO in these mutations and follows the FDA’s approval of KALYDECO in May 2017 for 23 other residual function mutations, which was based on analyses of in vitro data. Both approvals are supported by more than five years of real-world clinical experience that demonstrate KALYDECO’s established safety and efficacy profile. Based on today’s approval, Vertex increased its guidance for 2017 KALYDECO product revenues to a range of $770 million to $800 million. Vertex’s guidance range for total CF product revenues in 2017 is now $1.87 billion to $2.1 billion, including ORKAMBI guidance of $1.1 billion – $1.3 billion.

“In the five years since KALYDECO became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease.”

CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt (sodium and chloride) and water into or out of cells in a number of organs, including the lungs. The five mutations covered under today’s approval (2789+5G— > A, 3272-26A— > G, 3849+10kbC— > T, 711+3A— > G, and E831X) cause CF and result in a moderate loss of chloride transport. People who have these mutations generally experience progressive lung function decline and other complications of the disease. All five of these mutations were evaluated as part of the previously disclosed Phase 3 EXPAND study in which the KALYDECO monotherapy arm met its primary efficacy endpoint and was generally well tolerated.

KALYDECO is now approved in the U.S. to treat people with CF ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.

Shares of Vertex closed yesterday at $151.82, down $2.33 or -1.51%. VRTX has a 1-year high of $167.86 and a 1-year low of $71.46. The stock’s 50-day moving average is $136.85 and its 200-day moving average is $111.45.

On the ratings front, VRTX has been the subject of a number of recent research reports. In a report issued on July 27, Oppenheimer analyst Hartaj Singh reiterated a Buy rating on VRTX, with a price target of $175, which implies an upside of 15% from current levels. Separately, on the same day, Maxim Group’s Gabrielle Zhou reiterated a Buy rating on the stock and has a price target of $195.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Hartaj Singh and Gabrielle Zhou have a yearly average return of 10.9% and a loss of 0.6% respectively. Singh has a success rate of 58% and is ranked #834 out of 4627 analysts, while Zhou has a success rate of 43% and is ranked #3446.

Sentiment on the street is mostly bullish on VRTX stock. Out of 18 analysts who cover the stock, 16 suggest a Buy rating and 2 recommend to Hold the stock. The 12-month average price target assigned to the stock is $168.67, which implies an upside of 11% from current levels.

Vertex Pharmaceuticals, Inc. engages in the business of discovering, developing, manufacturing and commercializing small molecule drugs for patients with serious diseases. It focuses on development and commercializing therapies for the treatment of cystic fibrosis; infectious diseases, including viral infections, such as influenza, and bacterial infections; autoimmune diseases, such as rheumatoid arthritis; cancer, inflammatory bowel disease; and neurological disorders, including pain, Huntington’s disease and multiple sclerosis. 

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