- 12 abstracts presented at 30th Annual North
American Cystic Fibrosis Conference highlight data from Vertex’s CF
program -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the presentation of long-term data demonstrating that
ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) show the
potential to modify the progression of cystic fibrosis (CF). The
presentations given at the 30th Annual North American Cystic
Fibrosis Conference (NACFC) include final data from the PROGRESS
96-week extension study of the pivotal 24-week Phase 3 studies of
ORKAMBI in people ages 12 and older with two copies of the F508del
mutation (TRAFFIC and TRANSPORT). Other highlights include an
analysis of real-world outcomes in patients treated with KALYDECO
using data from the United States Cystic Fibrosis Foundation
Patient Registry (U.S. CFFPR) and United Kingdom Cystic Fibrosis
Registry (U.K. CFR). Vertex will webcast an investor presentation
from the conference at approximately 6:30 p.m. ET on Thursday,
October 27 that will provide an overview of the company’s progress
to develop more medicines to treat the underlying cause of CF in
all people with this disease. The webcast can be accessed live
through Vertex’s website.
“The growing body of long-term data for KALYDECO and ORKAMBI
indicates that treating the underlying cause of CF with CFTR
modulators may modify the progression of this serious and
life-shortening disease,” said Jeffrey Chodakewitz, M.D., Executive
Vice President and Chief Medical Officer at Vertex. “These data
support our goal to develop increasingly effective combination
regimens of CFTR modulators for all people with CF.”
The 12 related abstracts presented at NACFC include data from
studies of Vertex medicines and medicines in development.
Highlights include:
Real-World Outcomes of Ivacaftor Treatment
Analysis of Real-World Outcomes in Patients with CF Treated
with Ivacaftor from the 2014 U.S. and U.K. CF Registries
Abstract #494, Workshop 23: Epidemiology of CF, Saturday,
October 29, 10:56 a.m. to 11:06 a.m.
and
Analysis of Disease Progression in Patients with CF Treated
with Ivacaftor in the Real World Using Data From the U.K.CF
Registry
Abstract #495; Poster Session 1, Thursday, Oct. 27, 11:15 a.m.
to 1:45 pm; Basic Science: Friday, Oct. 28, 4:00 p.m. to 6:00
p.m.
Interim data from the ongoing, five-year, post-approval
observational safety study evaluating long-term outcomes in CF
patients treated with ivacaftor showed the potential of ivacaftor
to modify the progression of CF disease in a real-world setting. In
total, these analyses included patients who had received ivacaftor
for up to five years including 1,256 patients from the U.S. CFFPR
who received an average of two years of treatment and 411 patients
from the U.K. CFR who received an average of 1.3 years of
treatment; and untreated patients with differing genotypes,
including 6,200 from the U.S. CFFPR and 2,069 from the U.K. CFR. In
the U.S. registry, the annual risks of death, transplantation,
hospitalization and pulmonary exacerbation were each statistically
significantly lower compared to the comparator cohort of matched
patients who never received ivacaftor. Trends were similar in the
U.K. registry, but the differences in the risk of death and
transplantation were not statistically significant. No new safety
concerns were identified, and the majority of the CF-related
complications, such as CF-related diabetes and cultures positive
for several microbial pathogens, were less common among
ivacaftor-treated than untreated patients in both the U.S. and U.K.
registries.
Evidence of Reduction in Annual Rate of FEV1
Decline and Sustained Benefits with Lumacaftor and Ivacaftor in
Patients with Cystic Fibrosis Homozygous for F508del-CFTR
Abstract #180, Workshop 07: Clinical Advances in CF Research,
Thursday, Oct. 27, 10:50 a.m. to 11:00 a.m. ET
An analysis of 96-week data from the PROGRESS extension study of
the pivotal Phase 3 TRAFFIC and TRANSPORT studies of
lumacaftor/ivacaftor in people ages 12 and older with two copies of
the F508del mutation showed that lumacaftor/ivacaftor was generally
well tolerated and had a safety profile consistent with that seen
in TRAFFIC and TRANSPORT. In this 96-week safety extension study,
all participants received one of two lumacaftor/ivacaftor
combination regimens (400mg lumacaftor q12h in combination with
250mg ivacaftor q12h, or 600mg lumacaftor QD in combination with
ivacaftor 250mg q12h). Mean lung function, a secondary endpoint of
PROGRESS, was maintained above TRAFFIC/TRANSPORT baseline for up to
120 weeks.
An analysis of the rate of lung function decline was also
conducted at week 96 of PROGRESS to compare the results for people
who received the approved commercial dose of lumacaftor (400mg
q12h) in combination with ivacaftor (250mg q12h) (n=455) to a
propensity score matched cohort (n=1,588) of patients from the U.S.
CFFPR. The analysis showed that lumacaftor/ivacaftor reduced the
estimated annual rate of lung function decline by approximately 42
percent compared with matched controls. The annual rate of lung
function decline among those receiving lumacaftor/ivacaftor was
-1.33 percentage points compared to the matched control group,
which had a rate of decline of -2.29 percentage points per year
(p<0.001).
The most common adverse events occurring in ≥20 percent of
patients in either treatment group were pulmonary exacerbation,
cough, sputum increased, hemoptysis, and dyspnea. Mean blood
pressure increased from 113.4/68.7 mmHg at baseline of
TRAFFIC/TRANSPORT to 118.0/72.8 mmHg at week 96 of PROGRESS in
patients continuing lumacaftor 400 mg q12h/ivacaftor and increased
from 113.2/68.6 mmHg at baseline of TRAFFIC/TRANSPORT to 119.1/73.5
mmHg at week 96 of PROGRESS in patients who transitioned from
placebo to lumacaftor 400 mg q12h/ivacaftor.
“These data suggest that the benefits of lumacaftor/ivacaftor
are sustained through 96 weeks and indicate that the medicine may
modify the progression of CF lung disease by treating its
underlying cause,” said Michael W. Konstan, M.D., Vice Dean for
Translational Research at Case Western Reserve University School of
Medicine and Vice Chair for Clinical Research at University
Hospitals Rainbow Babies & Children’s Hospital who was the
principal investigator of the study.
Safety, Tolerability, and Pharmacodynamics of Combination
Lumacaftor/Ivacaftor Therapy in Patients Aged 6-11 years with CF
Homozygous for the F508del-CFTR mutation
Abstract 179; Workshop 07: Clinical Advances in CF Research,
Thursday, Oct. 27, 10:35 a.m. to 10:45 a.m. ET
Final data from an open-label Phase 3 safety study that
evaluated ORKAMBI in children with CF ages 6 through 11 who have
two copies of the F508del mutation showed that lumacaftor/ivacaftor
was generally well tolerated. The primary endpoint was safety. No
new safety events were observed compared to older patients.
Respiratory events (dyspnea, wheezing, respiration abnormal) were
infrequent and not associated with any discontinuations. In the
study, all children received a twice-daily fixed-dose combination
of lumacaftor (200mg) and ivacaftor (250mg) for 24 weeks. These
data supported the recent U.S. Food & Drug Administration
approval of lumacaftor/ivacaftor in children ages 6 through 11 who
have two copies of the F508del mutation.
Long-term Safety and Efficacy of Ivacaftor in Pediatric
Patients Aged 2-5 years with CF and a CFTR Gating Mutation
Abstract 177; Workshop 07: Clinical Advances in CF Research,
Thursday, Oct. 27, 10:05 a.m. to 10:15 a.m.
Data from the KLIMB and KIWI studies of ivacaftor in children
ages 2 through 5 with CF and a CFTR gating mutation showed that
ivacaftor demonstrated a durable safety profile for a total of 108
weeks of continuous treatment. Sweat chloride reductions in the
24-week open label Phase 3 KIWI study were maintained through
KLIMB, an 84-week open-label extension study. Age-normalized growth
parameters were either maintained or improved from KIWI baseline
through KLIMB. The most common adverse event of any grade was
cough. Elevated alanine aminotransferase (ALT) and aspartate
aminotransferase (AST) levels >3x the upper limit of normal
(ULN) were reported in ten out of 33 patients. Of these ten, four
had elevated ALT/AST levels of >8x ULN in KIWI. Ivacaftor was
maintained or resumed in all but one patient who discontinued and
elevated ALT/AST levels resolved upon discontinuation.
About the Rate of Lung Function Decline Analysis
PROGRESS is the 96-week extension study of TRAFFIC and
TRANSPORT, the pivotal 24-week Phase 3 studies of ORKAMBI in people
ages 12 and older with two copies of the F508del mutation. For the
rate of lung function decline analysis, a propensity score approach
was used to match patients from PROGRESS to patients from the U.S.
CFFPR on known predictors of lung function decline. Propensity
scoring is a statistical matching technique used in observational
research that attempts to balance the study groups to make them as
similar as possible. The analysis compared 455 patients who
received ORKAMBI for up to 96 weeks and observational data from the
U.S. CFFPR on 1,588 control patients homozygous for the F508del
mutation. Estimates of average annual rate of decline were based on
lung function measurements spanning different lengths of time for
different patients, with more patients contributing information
about the rate of decline in the first year than in the second
year.
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.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO®
(ivacaftor)
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 2 years and older
who have one of the following mutations in their CF gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or
R117H. KALYDECO is not for use in people with CF due to other
mutations in the CF gene. KALYDECO is not effective in patients
with CF 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 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 or Seville oranges; are pregnant or plan to become
pregnant because it is not known if KALYDECO will harm an unborn
baby; and are breastfeeding or planning to breastfeed because is
not known if KALYDECO passes into breast milk.
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 Seville oranges
while taking KALYDECO.
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 (ivacaftor).
About Cystic Fibrosis
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.
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.
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. KALYDECO
(ivacaftor) and ORKAMBI (lumacaftor/ivacaftor) were discovered by
Vertex as part of this collaboration.
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 from Dr. Chodakewitz
and Dr. Konstan. 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 data from the
company's development programs may not support registration or
further development of ORKAMBI, KALYDECO or its other 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)
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Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia: mediainfo@vrtx.comNorth
America:Chris Stamm, +1 617-341-6992orEurope & Australia:Megan
Goulart, +44 20 3204 5275
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