-Real-world data presented at ECFS show
long-term impact of KALYDECO across multiple measures of
disease-
-Data from Phase 3 safety study of ORKAMBI in
children ages 6-11 presented today at ECFS-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced presentations of KALYDECO® (ivacaftor) and ORKAMBI®
(lumacaftor/ivacaftor) data at the 39th European Cystic Fibrosis
Society (ECFS) Conference, being held June 8 to 11, 2016 in Basel,
Switzerland. The presentations include new real-world data from an
ongoing five-year observational study evaluating long-term outcomes
in cystic fibrosis (CF) patients treated with ivacaftor, as well as
results from the Phase 3 safety study of lumacaftor/ivacaftor in
children ages 6 through 11 with CF.
“Our scientists have been working for many years to change the
way CF is treated by developing medicines that address the
underlying cause of the disease,” said Jeffrey Chodakewitz, M.D.,
Executive Vice President and Chief Medical Officer at Vertex. “As
more data for KALYDECO and ORKAMBI become available, we are
increasingly confident that treating the underlying cause of the
disease slows its progression and results in a range of benefits
across multiple measures of CF. We’re committed to continuing our
efforts to help the two out of three people with CF who don’t
currently have a medicine that treats the underlying cause of their
disease.”
Real-world outcomes in people with cystic fibrosis treated
with ivacaftor
New interim data from the ongoing, five-year, post-approval
observational safety study evaluating long-term outcomes in CF
patients treated with ivacaftor in a real-world setting were
presented this week. The following ECFS presentations are based on
the third annual analysis conducted as part of this study, which
uses data collected by the U.S. CF Foundation Patient Registry and
the U.K. CF Registry through 2014:
- “Disease progression in patients with
cystic fibrosis treated with ivacaftor: analysis of real-world data
from the U.K. CF Registry.” ePoster ePS03.1.
- “Real-world outcomes in patients with
cystic fibrosis treated with ivacaftor: analysis of 2014 U.S. and
U.K. registries.” ePoster ePS03.2.
- “Real-world outcomes in young (aged 6
to 12 years) patients with cystic fibrosis treated with ivacaftor:
analysis of 2014 U.S. and U.K. CF registries data.” Poster 25.
In total, these analyses included patients who had received
ivacaftor for up to five years including 1,256 patients from the
U.S. registry who received an average of two years of treatment and
411 patients from the U.K. registry who received an average of 1.3
years of treatment. In the U.S. registry, the annual risks of
death, transplantation, hospitalization and pulmonary exacerbation
were significantly lower than in 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. Long-term follow-up data from both registries indicate
clinically important outcomes in patients treated with ivacaftor in
real-world settings across multiple measures of CF that are
indicative of disease modification.
Phase 3 safety study of lumacaftor/ivacaftor in children ages
6 to 11
Final data were presented from an open-label Phase 3 safety
study that evaluated lumacaftor/ivacaftor in 58 children with CF
ages 6 through 11 who have two copies of the F508del mutation. In
the study, all children received a twice-daily fixed-dose
combination of lumacaftor (200mg) and ivacaftor (250mg) for 24
weeks. As announced in January 2016, the study met its primary
safety endpoint.
Safety data showed that the combination was well tolerated, and
the most common adverse events were cough, headache, infective
pulmonary exacerbation, nasal congestion, abdominal pain, increased
sputum and elevated liver enzymes. Two patients (3.4%) discontinued
treatment because of adverse events (rash and abnormal liver
function tests). Respiratory events, including dyspnea, respiration
abnormal and wheezing, were observed in four patients (6.9%) and
were not associated with treatment discontinuation. Serious adverse
events were reported in four patients (6.9%), with one patient
(1.7%) having elevated abnormal liver function tests.
Additional details from the study are being presented today at
ECFS, including results for the study’s secondary and exploratory
efficacy endpoints, as part of Workshop 19, Late Breaking
Science.
At 24 weeks, there were improvements in multiple secondary
endpoints, including a reduction in sweat chloride of -24.8 mmol/L
(p<0.0001), a weight gain of 2.6 kg (p<0.0001), an
improvement in the CFQ-R respiratory domain score of 5.4 points
(p=0.0085), an absolute improvement in FEV1 of 2.5 percentage
points (p=0.067), and a -0.88 (p=0.0018) improvement in the
exploratory endpoint of lung clearance index (LCI2.5).
The U.S. FDA recently granted Vertex's request for Priority
Review of a supplemental New Drug Application (sNDA) for approval
of ORKAMBI for children ages 6 through 11 who have two copies of
the F508del mutation and set a target review date of September 30,
2016 for a decision on the sNDA. There are approximately 2,400
children ages 6 to 11 who have two copies of the F508del mutation
in the U.S.
Enrollment is complete in a six-month Phase 3 efficacy study
required to support potential approval of lumacaftor/ivacaftor in
the European Union in children ages 6 through 11 who have two
copies of the F508del mutation. The study is evaluating
lumacaftor/ivacaftor in approximately 200 children and the primary
endpoint is the absolute change in lung clearance index. Pending
data from the study, Vertex plans to submit a Marketing
Authorization Application (MAA) variation in the European Union in
the first half of 2017. In Europe, there are approximately 3,400
children ages 6 through 11 who have two copies of the F508del
mutation.
Efficacy and safety of lumacaftor/ivacaftor across sub-groups
in TRAFFIC and TRANSPORT studies
A pre-specified pooled analysis of the TRAFFIC and TRANSPORT
studies evaluated whether baseline lung function was predictive of
the efficacy and safety of lumacaftor/ivacaftor treatment by
stratifying patients based on their screening and study baseline
lung function values. These data were previously presented at the
North American CF Conference in October 2015 and will be presented
as part of an invited talk during Symposium 29, Best of Journal of
Cystic Fibrosis / The Lancet Respiratory Medicine Symposium, at
ECFS.
About ORKAMBI® (lumacaftor/ivacaftor)
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. It is an oral
pill taken every 12 hours - once in the morning and once in the
evening.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About KALYDECO® (ivacaftor)
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, ivacaftor is an oral medicine that aims to help
the CFTR protein function more normally once it reaches the cell
surface, to help hydrate and clear mucus from the airways.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease
affecting approximately 75,000 people in North
America, Europe and Australia. Today, the median
predicted age of survival for a person with CF is between 34 and 47
years, but the median age of death remains in the mid-20s.
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 more than 1,900 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 protein at the cell surface. The
defective function or absence of CFTR proteins in people with CF
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.
Collaborative History with Cystic Fibrosis Foundation
In 1998, Vertex initiated its CF research program in connection
with its collaborative relationship with the Cystic Fibrosis
Foundation. KALYDECO® (ivacaftor) and ORKAMBI®
(lumacaftor/ivacaftor) were discovered by Vertex as part of this
collaboration.
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.
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, Dr. Chodakewitz's statements in the
second paragraph of the press release, the target review date of
September 30, 2016 for a decision on the sNDA for approval of
ORKAMBI for children ages 6 to 11 who have two copies of the
F508del mutation and the expected timing for submission of an MAA
variation in Europe for children ages 6 to 11 who have two copies
of the F508del mutation. While Vertex 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, that regulatory authorities may not approve, or approve on
a timely basis, the sNDA, 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 the 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,
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617-341-6470orMedia:mediainfo@vrtx.comEurope & Australia:Megan
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617-341-6992
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