ORKAMBI Financial GuidanceVertex today revised its guidance for 2016 ORKAMBI revenues. The company now expects ORKAMBI revenues of $950 million to $990 million. Vertex's prior guidance was for total 2016 ORKAMBI revenues of $1.0 billion to $1.1 billion, and to date Vertex has reported ORKAMBI revenues of $223 million and $245 million for the first and second quarters of 2016, respectively. Vertex expects third quarter ORKAMBI revenues to be between $230 million and $235 million. The revised guidance primarily reflects the following:
- The slower than anticipated launch in Germany where fewer than 20 percent of the approximately 2,500 eligible patients have initiated treatment to date;
- That we are approaching peak penetration for ORKAMBI in the U.S. where there are approximately 8,500 eligible patients ages 12 and older; and
- Slower than expected refills for ORKAMBI during the summer months of July and August.
When taking ORKAMBI, patients should tell their doctor if they stop ORKAMBI for more than 1 week as the doctor may need to change the dose of ORKAMBI or other medicines the patient is taking. It is unknown if ORKAMBI causes dizziness. Patients should not drive a car, use machinery, or do anything requiring alertness until the patient knows how ORKAMBI affects them.ORKAMBI can cause serious side effects including: High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI. The patient's doctor will do blood tests to check their liver before they start ORKAMBI, every three months during the first year of taking ORKAMBI, and annually thereafter. The patient should call the doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine; or confusion. Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If a patient has poor lung function, their doctor may monitor them more closely when starting ORKAMBI. An increase in blood pressure has been seen in some patients treated with ORKAMBI. The patient's doctor should monitor their blood pressure during treatment with ORKAMBI. Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI. For children and adolescents, the patient's doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts. The most common side effects of ORKAMBI include: shortness of breath and/or chest tightness; upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose; gastrointestinal symptoms including nausea, diarrhea, or gas; rash; fatigue; flu or flu-like symptoms; increase in muscle enzyme levels; and irregular, missed, or abnormal menstrual periods and heavier bleeding.
Please click here to see the full Prescribing Information for ORKAMBI.Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. ORKAMBI (lumacaftor/ivacaftor) was discovered by Vertex as part of this collaboration. About Vertex Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases. Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia. For six years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. For additional information and the latest updates from the company, please visit www.vrtx.com. Special Note Regarding Forward-looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided in Dr. Chodakewitz's quote in the second paragraph of this press release and in the section captioned "ORKAMBI Financial Guidance" and statements regarding (i) Vertex's revenue guidance and expectations and (ii) plans to submit an MAA variation in the European Union for children ages 6 to 11 who have two copies of the F508del mutation and the timing of expected data from Vertex's Phase 3 efficacy study in this patient population. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding revenues may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)