Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of ORKAMBI® (lumacaftor/ivacaftor) in people with cystic fibrosis (CF) ages 6 to 11 who have two copies of the F508del mutation. The FDA granted Vertex’s request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act (PDUFA).
“As complications related to CF can occur early in life, we believe it is important to begin treatment as early as possible and this supplemental New Drug Application for approval of ORKAMBI in children as young as six is an important step in that direction,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “This submission demonstrates Vertex’s continued progress toward our goal of developing medicines for all people with CF.”
The sNDA is based on data from an open label Phase 3 clinical safety study of ORKAMBI. Data from this study will be presented at the 39th European Cystic Fibrosis Society conference being held June 8 to 11 in Basel, Switzerland.
To support potential approval in the European Union, a six-month Phase 3 efficacy study of children ages 6 to 11 is ongoing. Vertex recently completed enrollment in this study and, pending data from the study, plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children ages 6 to 11 who have two copies of the F508del mutation. The primary endpoint of this efficacy study is the absolute change in lung clearance index (LCI). There are approximately 3,400 children ages 6 to 11 who have two copies of the F508del mutation in the European Union. (Original Source)
Shares of Vertex Pharmaceuticals are currently trading at $92.86, up $2.48 or 2.74%. VRTX has a 1-year high of $143.45 and a 1-year low of $75.90. The stock’s 50-day moving average is $84.66 and its 200-day moving average is $97.15.
On the ratings front, Vertex has been the subject of a number of recent research reports. In a report issued on May 27, Jefferies Co. analyst Brian Abrahams reiterated a Buy rating on VRTX, with a price target of $103, which represents a potential upside of 13.7% from where the stock is currently trading. Separately, on May 12, Morgan Stanley’s Matthew Harrison reiterated a Buy rating on the stock and has a price target of $137.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Brian Abrahams and Matthew Harrison have a total average return of 12.1% and 4.8% respectively. Abrahams has a success rate of 62.1% and is ranked #227 out of 3891 analysts, while Harrison has a success rate of 58.1% and is ranked #962.
Overall, 6 research analysts have assigned a Hold rating and 9 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $112.80 which is 24.5% above where the stock opened today.
Vertex Pharmaceuticals, Inc. engages in the business of discovering, developing, manufacturing, and commercializing small molecule drugs for patients with serious diseases in specialty markets. 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.