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FDA Approves KALYDECO® (ivacaftor) for More Than 900 People Ages 2 and Older with Cystic Fibrosis Who Have Certain Residual Function Mutations
- Precision medicine decision based on in vitro data and supported by more than five years of real-world clinical data that demonstrate KALYDECO's strong safety and efficacy profile for eligible patients -
- Vertex working with
In addition to the mutations added to the label today, Vertex is
continuing discussions with the
"Five years ago, KALYDECO became the first medicine to treat the
underlying cause of CF. Since then, we have continued to invest in
studies to improve the understanding of how this important medicine may
benefit others with this serious and life-shortening disease," said
"CF treatment has advanced rapidly, but there is need for broader access
to these important medicines and development of additional medicines
remains urgent," said
Based on clinical data from numerous prior studies and a demonstrated in vitro response employing a well-established, analytically validated method, KALYDECO is now approved to treat people with CF ages 2 and older who have one of 33 mutations in the CFTR gene.
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 (chloride) and water into and out of the cell in a number of organs, including the lungs. Chloride transport is a marker of the function of the CFTR protein at the cell surface. As with the other mutations for which KALYDECO is approved, mutations covered under today's approval are known to have some CFTR protein at the cell surface and have shown in vitro increases in chloride transport in response to KALYDECO by at least 10 percent of normal over baseline. Similar to the R117H mutation for which KALYDECO was previously approved, these additional residual function mutations result in a moderate loss of CFTR chloride transport, and people with these mutations generally have progressive lung function decline and other complications of CF.
Pipeline Progress for Residual Function Mutations
About Cystic Fibrosis and KALYDECO® (ivacaftor)
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing CFTR protein 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 is 41 years, but the median age of death is 27 years.
KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. In adults and pediatric patients age 6 years and older one 150 mg tablet is taken orally every 12 hours with fat-containing food. Pediatric patients ages 2 to less than 6 years who weigh less than 14 kg take one 50 mg packet of oral granules mixed with 1 teaspoon (5 mL) of soft food or liquid administered orally every 12 hours with fat-containing food. Pediatric patients ages 2 to less than 6 years of age who weigh 14 kg or greater take one 75 mg packet of oral granules with 1 teaspoon (5 mL) of soft food or liquid administered orally every 12 hours with fat-containing food.
People with CF who have specific mutations in the CFTR gene are
currently benefiting from KALYDECO in 27 different countries across
KALYDECO® (ivacaftor) INDICATION AND IMPORTANT SAFETY INFORMATION
KALYDECO (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have at least one mutation in the CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 2 years of age.
Patients should not take KALYDECO if they are taking certain medicines or herbal supplements such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.
Before taking KALYDECO, patients should tell their doctor if they:
have liver or kidney problems; drink grapefruit juice, or eat grapefruit
KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Therefore the dose of KALYDECO may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it. Patients should
not drive a car, use machinery, or do anything that needs them to be
alert until they know how KALYDECO affects them. Patients should avoid
food containing grapefruit or
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in patients receiving KALYDECO. The patient's doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often. Patients should call their 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 their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. The patient's doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts. The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing Information for KALYDECO.
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
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 statements in the third and fourth paragraphs of the
press release and statements regarding (i) the potential approval of
KALYDECO for patients with "splice" mutations, (ii) Vertex's updated
guidance for KALYDECO 2017 net product revenues and (iii) Vertex's plans
to submit an NDA and MAA for tezacaftor/ivacaftor. 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 its 2017 KALYDECO net product 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
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