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FDA Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis
- KALYDECO approved to treat people with CF who have a specific genetic mutation -
- Vertex launches a comprehensive financial assistance and patient support program -
KALYDECO carton, bottle, and pills (Photo: Business Wire)
Vertex is ready to support the introduction of KALYDECO and will begin
shipping it to pharmacies in
"More than 13 years ago we set out to change the lives of people with
cystic fibrosis by developing new medicines that address the underlying
cause of this rare and devastating disease," said
The approval of KALYDECO was based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain and certain quality of life measurements, compared to those who received placebo. People who took KALYDECO also experienced significantly fewer pulmonary exacerbations, which are periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of adverse events associated with KALYDECO were mild to moderate. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea.
"Advances in cystic fibrosis treatment have helped manage symptoms of
the disease, however people with cystic fibrosis still have a hard time
staying healthy and being active," said
"Together, we're changing the lives of people with cystic fibrosis,"
Cystic fibrosis is a rare, life-threatening genetic disease for which there is no cure. CF is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that control the flow of salt and water across the cells. When the defective CFTR protein does not work properly at the cell surface, abnormally thick, sticky mucus builds up in the lungs. The digestive tract and a number of other organs are also affected. KALYDECO, an oral medicine known as a CFTR potentiator, helps the CFTR protein function more normally once it reaches the cell surface. KALYDECO targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways, and keeps them from getting clogged and then infected.
Because KALYDECO targets a specific genetic mutation, a person's
genotype should be known before this new medicine is prescribed. Genetic
testing is widely available and
KALYDECO by itself works in a subset of people with CF, but research is ongoing to explore a similar targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease.
Helping People with CF Get KALYDECO
The people who work at Vertex understand that medicines can only help patients who can get them. To that end, the company offers a comprehensive financial assistance and patient support program. A specially-trained and dedicated Vertex team will provide one-on-one support to help eligible patients who are prescribed KALYDECO understand their insurance benefits and the resources that are available to help them.
For eligible patients, the program also includes the following:
Free Medicine Program: Vertex will provide KALYDECO for free to people
who do not have insurance and have an annual household income of
$150,000or less; and
Co-Pay Assistance Program: For patients with commercial insurance plans that cover KALYDECO and who are enrolled in the Guidance and Patient Support, or GPS, program, there will be a minimal out-of-pocket obligation after which Vertex will help cover co-pay or co-insurance costs up to 30 percent of the list price of the medicine. There is no income limit to be eligible for this program.
Some patients are not eligible for company co-pay support because they
More information about this program is available by calling 1-877-7-KALYDECO (877-752-5933) or visiting www.VertexGPS.com.
KALYDECO is the first treatment to target the underlying cause of CF. The Phase 3 studies evaluated KALYDECO in people with CF ages 6 and older who had at least one copy of the G551D mutation. PERSIST, a Phase 3, open-label, 96-week extension study, is underway to evaluate the long-term safety and durability of treatment with KALYDECO. This ongoing study enrolled people who completed 48 weeks of treatment in either Phase 3 study (placebo and KALYDECO treatment groups) and met other eligibility criteria. KALYDECO will be taken as one 150-mg tablet twice daily (every 12 hours).
Vertex retains worldwide rights to develop and commercialize KALYDECO.
Indication and Important Safety Information
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have a certain mutation in their CF gene called the G551D mutation.
KALYDECO is not for use in people with CF due to other mutations in the CF gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CF 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. High liver enzymes in the blood have occurred in patients taking KALYDECO. 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 Prescribing Information for KALYDECO at www.KALYDECO.com.
Conference Call for Media and Investors
Vertex will host a conference call and webcast today,
To listen to the live call on the telephone, dial 1-877-250-8889 (
The conference ID number for the live call and replay is 48426093.
The call will be available for replay via telephone commencing
Following the live webcast, an archived version will be available on
Vertex's website until
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 30,000 people in
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. Approximately 4 percent of those with CF, or about 1,200
In people with the most common mutation in the CFTR gene, F508del, the CFTR protein does not reach the cell surface in normal amounts and the CFTR proteins that reach the surface do not work correctly. Nearly 90 percent of people with CF have at least one copy of the F508del mutation; approximately half of those with CF have two copies. KALYDECO is not effective in CF patients who have two copies of the F508del mutation in the CFTR gene.
KALYDECO has been approved by the
Enrollment is ongoing in the second part of a Phase 2 clinical trial of combination regimens of KALYDECO and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. In addition, the company plans to begin Phase 2 development of VX-661, a second CFTR corrector, in the first quarter of 2012.
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, as amended,
including statements regarding (i) Vertex being ready to support the
introduction of KALYDECO and beginning to ship it to pharmacies this
week; (ii) Vertex's financial assistance and patient support programs;
(iii) the progress Vertex is making toward its ultimate goal of
developing additional medicines to help many more people with cystic
fibrosis; (iv) the roadmap provided by KALYDECO for exploring additional
targeted approaches to treatment for all people with cystic fibrosis;
(v) the ongoing research to explore a targeted approach using a
combination of medicines, including KALYDECO, to treat the most common
form of the disease and (vi) planned additional clinical trials of
KALYDECO in children as young as 2 years old and people with CF who have
the R117H mutation and gating mutations that were not evaluated in
previous Phase 3 clinical trials. While the company believes the
forward-looking statements contained in this press release are accurate,
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,
risks related to the commercialization of KALYDECO and development of
additional medicines to treat cystic fibrosis and the other risks listed
under Risk Factors in Vertex's annual report and quarterly reports filed
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