Press Release
U.S. Food and Drug Administration Approves ORKAMBI® (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 through 11 have two copies of
the F508del mutation in the
- Vertex revises ORKAMBI revenue guidance for 2016 -
"The ability to treat children as young as six who have the most common
form of the disease is an important milestone as we pursue our goal to
develop medicines for all people with CF," said
The approval is based on data from a previously announced open-label
Phase 3 clinical safety study of ORKAMBI presented at the 39th
Vertex plans to submit a Marketing Authorization Application (MAA)
variation in the
ORKAMBI Financial Guidance
Vertex today revised its guidance for 2016 ORKAMBI revenues. The company
now expects ORKAMBI revenues of
-
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.
Today's approval in people ages 6 through 11 will drive growth in the
About Cystic Fibrosis and ORKAMBI
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by defective or missing cystic fibrosis conductance
regulator (CFTR) proteins resulting from mutations in the CFTR gene.
Children must inherit two defective CFTR genes — one
from each parent — to have CF. There are approximately 2,000 known
mutations in the CFTR gene. Some of these mutations,
which can be determined by a genetic test, lead to CF by creating
defective or too few CFTR proteins at the cell surface. The defective or
missing CFTR protein results in poor flow of salt and water into or out
of the cell in a number of organs, including the lungs. This leads to
the buildup of abnormally thick, sticky mucus that can cause chronic
lung infections and progressive lung damage in many patients that
eventually leads to death. The median predicted age of survival for a
person born today with CF in
In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little to no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. In pediatric patients ages 6 through 11, two ORKAMBI tablets (each containing lumacaftor 100mg/ivacaftor 125mg) are taken orally every 12 hours - once in the morning and once in the evening - with fat-containing food.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI® (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should only be used in these patients. It is not known if ORKAMBI is safe and effective in children under 6 years of age.
Patients should not take ORKAMBI if they are taking certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; the seizure medicines phenobarbital, carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines triazolam or midazolam; the immunosuppressant medicines everolimus, sirolimus, or tacrolimus; or St. John's wort.
Before taking ORKAMBI, patients should tell their doctor if they: have or have had liver problems; have kidney problems; have had an organ transplant; are using birth control (hormonal contraceptives, including oral, injectable, transdermal or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI. Patients should tell their doctor if they are pregnant or plan to become pregnant (it is unknown if ORKAMBI will harm the unborn baby) or if they are breastfeeding or planning to breastfeed (it is unknown if ORKAMBI passes into breast milk).
ORKAMBI may affect the way other medicines work and other medicines may affect how ORKAMBI works. Therefore, the dose of ORKAMBI or other medicines may need to be adjusted when taken together. Patients should especially tell their doctor if they take: antifungal medicines such as ketoconazole, itraconazole, posaconazole, or voriconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
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
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the
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
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
(VRTX-GEN)
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