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Data From Follow-Up Study of KALYDECO™ (ivacaftor) Showed Durable Improvements in Lung Function and Other Measures of Disease in People with Cystic Fibrosis Who Have a Specific Genetic Mutation (G551D)
- Data from nine presentations at the
"KALYDECO has fundamentally changed the way we approach the development
of new medicines for cystic fibrosis by targeting the underlying cause
of the disease," said
KALYDECO is the first medicine to treat the underlying cause of CF, a
rare, genetic disease caused by defective or missing CFTR proteins
resulting from mutations in the CFTR gene. In people with the
G551D mutation, KALYDECO helps the defective CFTR protein function more
normally. An estimated 1,200 people in
KALYDECO benefits and safety sustained for up to 96 weeks (ECFS
Abstract WS6.4, Thursday @
At the time of this analysis, 74 adults and adolescents (ages 12 or older) who were first treated with KALYDECO in the STRIVE study are continuing treatment in PERSIST and have completed a total of 96 weeks of treatment with KALYDECO. A 9.5 percentage-point mean absolute improvement from the STRIVE baseline in lung function (percent predicted FEV1) was observed at week 96. Twenty-five children (ages 6 to 11) who were first treated with KALYDECO in the ENVISION study are continuing treatment in PERSIST and have completed a total of 72 weeks of treatment with KALYDECO. A 10.1 percentage-point mean absolute improvement from the ENVISION baseline in lung function was observed at week 72.
In addition, the analysis presented this week showed that people who switched to KALYDECO after receiving 48 weeks of treatment with placebo in the Phase 3 studies (n=86), experienced improvements in lung function, respiratory symptoms and weight gain comparable to those seen in people who received KALYDECO from the beginning of the Phase 3 studies.
Adverse events seen to date in people receiving KALYDECO in the PERSIST study were generally consistent with those seen with KALYDECO treatment during the original Phase 3 studies. The majority of adverse events associated with KALYDECO were mild or moderate in severity and resolved during the reporting period. No new adverse events were identified. The most common adverse events reported in PERSIST were predominantly respiratory-related and included pulmonary exacerbations, cough, productive cough and upper respiratory tract infection. The most commonly reported serious adverse events (that occurred in more than one patient) in PERSIST were pulmonary exacerbations, hemoptysis and intestinal obstruction. At the time of this PERSIST analysis, approximately 1.0 percent of study participants had discontinued treatment due to an adverse event.
KALYDECO improved lung function in people with early-stage CF (ECFS Abstract WS7.6, Thursday @18:00 GMT)
CF-related lung disease is known to start before it's detectable by deterioration in FEV1. Once FEV1 has fallen below normal, (80 percent to 85 percent predicted), structural damage may have already occurred; much of this can be irreversible.
Data from a Phase 2 randomized, double-blind, crossover study of people with early-stage CF (FEV1 greater than 90 percent predicted) ages 6 and older who have at least one copy of the G551D mutation, were presented at the conference and showed that KALYDECO led to statistically significant improvements in lung function. At baseline, the mean percent predicted FEV1 for study participants (n=20) was 97.2 percent. Through 29 days of treatment, the mean absolute improvement in lung function was 8.7 percentage points compared to placebo (p=0.0103).
"Cystic fibrosis is a progressive disease and as patients get older,
lung damage progresses and often becomes irreversible," said KALYDECO
KALYDECO significantly reduced risk of pulmonary exacerbations (ECFS Poster #44)
An analysis of data from the Phase 3 STRIVE study that enrolled people ages 12 and older with at least one copy of the G551D mutation was presented at the conference. As previously reported, people treated with KALYDECO were 55 percent less likely to experience a pulmonary exacerbation compared to those treated with placebo through week 48. Pulmonary exacerbations are generally considered periods of worsening in the signs and symptoms of CF that often require treatment with antibiotics and hospital visits. New data were obtained from statistical modeling and showed that patients treated with KALYDECO were also significantly less likely than those treated with placebo to require hospitalization and intravenous antibiotics. Through 48 weeks, people treated with KALYDECO, compared to those treated with placebo, were 67 percent less likely to require hospitalization for a pulmonary exacerbation and 59 percent less likely to require intravenous antibiotics for a pulmonary exacerbation.
Vertex continues to pursue goal of treating more people with CF
Vertex is committed to developing new medicines to treat the underlying cause of CF. The company plans to begin three additional pivotal studies in 2012 to explore the safety and efficacy of KALYDECO; a study of people with the R117H CFTR mutation, a study of people with CFTR gating mutations that were not evaluated in the previous Phase 3 studies, and a study of children with CF as young as 2 years old who have gating mutations. Vertex is also conducting two Phase 2 studies of KALYDECO in combination with a CFTR corrector, VX-809 or VX-661, to treat people with the most common form of CF.
KALYDECO, VX-809 and VX-661 were discovered as part of a collaboration
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease that affects
approximately 70,000 people worldwide, including 30,000 people in
Today, the median predicted age of survival for a person with CF is approximately 38 years but the median age of death remains in the mid-20s. There are more than 1,800 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The absence of working CFTR proteins results in poor flow of salt and water into and 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 some people, CFTR proteins are present at the cell surface but do not work properly. One type of this dysfunction is known as the G551D mutation.
KALYDECO™ (ivacaftor) is the first treatment to target the underlying
cause of CF. KALYDECO (150mg, q12h) was approved by the
Vertex retains worldwide rights to develop and commercialize KALYDECO.
Indication and Important Safety Information
KALYDECO (150mg, q12h) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a certain mutation in their CFTR gene called the G551D mutation.
KALYDECO is not for use in people with CF due to other mutations in the CFTR gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CFTR gene.
It is not known if KALYDECO is safe and effective in children under 6 years of age.
KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort.
KALYDECO can cause serious side effects. Serious side effects that may or may not be related to KALYDECO but which occurred more frequently in patients treated with KALYDECO included stomach (abdominal) pain, high liver enzymes in the blood, and low blood sugar. Regular assessment is recommended.
The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including 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. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com.
Collaborative History with
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the
Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.
Founded more than 20 years ago in
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 Dr. Wright's
statements in the second paragraph of this press release, Dr. Davies'
statements in the ninth paragraph of this press release, and statements
regarding (i) Vertex's commitment to change CF treatment by targeting
the underlying cause of the disease, (ii) Vertex's plan to begin three
additional pivotal studies in 2012 to explore the safety and efficacy of
KALYDECO and (iii) regulatory submissions in
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