-In Australia, approximately 1,000 people with
CF ages 12 and older have two copies of the F508del mutation-
-ORKAMBI reimbursement process already underway
in Australia-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the Therapeutic Goods Administration (TGA) of
Australia has approved ORKAMBI® 200/125 (lumacaftor 200mg and
ivacaftor 125mg), the first medicine to treat the underlying cause
of cystic fibrosis (CF) in people ages 12 and older who have two
copies of the F508del mutation. In Australia, there are
approximately 1,000 people with CF ages 12 and older who have two
copies of this mutation. The Australian reimbursement process for
ORKAMBI is already underway as part of the parallel regulatory and
reimbursement processes between the TGA and the Pharmaceutical
Benefits Advisory Committee (PBAC).
“We are pleased that the Therapeutic Goods Administration has
recognised the ‘meaningful clinical benefit’ offered by ORKAMBI,”
said Simon Bedson, Senior Vice President and International General
Manager for Vertex. “Today’s approval is an important step toward
making ORKAMBI available for eligible Australians who do not
currently have a medicine to treat the underlying cause of their
disease.”
The TGA approval is based on previously announced data from two
24-week global Phase 3 studies, TRAFFIC and TRANSPORT, and
additional interim 24-week data from the subsequent extension
study, PROGRESS, in people with CF ages 12 and older who have two
copies of the F508del mutation and were already being treated with
standard-of-care medicines. In the TRAFFIC and TRANSPORT studies,
which enrolled more than 1,100 patients and were the largest CF
studies ever conducted, those treated with the combination of
lumacaftor and ivacaftor experienced significant improvements in
lung function. Patients also experienced improvements in body mass
index (BMI) and reductions in pulmonary exacerbations (acute lung
infections), including those requiring hospitalisations and
intravenous antibiotic use. Interim data from PROGRESS showed that
these improvements were sustained through 48 total weeks of
treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks in PROGRESS).
In addition, the pattern and magnitude of response observed after
the initiation of combination treatment across all patients who
received placebo in TRAFFIC and TRANSPORT and subsequently received
a combination regimen in PROGRESS were similar to those seen among
patients who received a combination regimen in TRAFFIC and
TRANSPORT.
The combination of lumacaftor and ivacaftor was generally well
tolerated in all three studies. In TRAFFIC and TRANSPORT, the most
common adverse events included shortness of breath and/or chest
tightness, upper respiratory tract infection (common cold) and
gastrointestinal symptoms (including nausea, diarrhea, or gas). In
the extension study, the safety and tolerability results, including
the type and frequency of adverse events and serious adverse
events, were consistent with those observed in TRAFFIC and
TRANSPORT, and no new safety concerns were identified. Over 48
weeks, the most common adverse events were infective pulmonary
exacerbation, cough and increased sputum. The incidence of serious
adverse events during PROGRESS was generally similar to TRAFFIC and
TRANSPORT.
ORKAMBI 200/125 is indicated for the treatment of cystic
fibrosis in patients age 12 years and older who are homozygous
for the F508del mutation in the CFTR gene.
ORKAMBI is only for use in patients who possess two copies
of the F508del mutation in the CFTR gene. The safety and efficacy
of ORKAMBI in children aged less than 12 years have not
been established. ORKAMBI should be used with caution in patients
with advanced liver disease and only if the benefits are expected
to outweigh risks. If ORKAMBI is used in patients with advanced
liver disease, they should be closely monitored after the
initiation of treatment and the dose should be reduced. Assessment
of liver function tests is recommended before initiation, every 3
months during the first year of treatment, and annually thereafter.
For patients with a history of ALT, AST, or bilirubin elevations,
more frequent monitoring should be considered. Caution is
recommended when administering ORKAMBI to patients with severe
renal impairment (creatinine clearance less than or equal to 30
mL/min) or end-stage renal disease. Respiratory events (e.g., chest
discomfort, dyspnea, and respiration abnormal) were observed more
commonly in patients during initiation of ORKAMBI compared to those
who received placebo. Clinical experience in patients with percent
predicted FEV1 (ppFEV1) <40 is limited, and additional
monitoring of these patients is recommended during initiation of
therapy. Menstrual abnormalities (amenorrhoea, dysmenorrhoea,
menorrhagia, menstruation irregular, metrorrhagia, oligomenorrhoea,
and polymenorrhoea) were more common in ORKAMBI treated female
patients than in placebo. These menstrual abnormalities were more
frequent in the subset of female patients who were taking hormonal
contraceptives. Increased blood pressure has been observed in some
patients treated with ORKAMBI. Blood pressure should be monitored
periodically in all patients during treatment. Lumacaftor is a
strong inducer of CYP3A; administration of ORKAMBI may decrease
systemic exposure of medicinal products which are substrates of
CYP3A, which may decrease their therapeutic effect.
Co-administration with sensitive CYP3A substrates or CYP3A
substrates with a narrow therapeutic index is not recommended.
ORKAMBI may substantially decrease hormonal contraceptive exposure,
reducing effectiveness. Hormonal contraceptives, including oral,
injectable, transdermal, and implantable, should not be relied upon
as an effective method of contraception when co-administered with
ORKAMBI. Ivacaftor is a substrate of CYP3A4 and CYP3A5 isoenzymes;
use of ORKAMBI with strong CYP3A inducers, such as rifampicin,
significantly reduces ivacaftor exposure, which may reduce the
therapeutic effectiveness of ORKAMBI. Therefore, co-administration
with strong CYP3A inducers (e.g., rifampicin, St. John’s wort
[Hypericum perforatum]) is not recommended. Cases of non-congenital
lens opacities without impact on vision have been reported in
paediatric patients treated with ivacaftor monotherapy. Baseline
and follow-up ophthalmological examinations are recommended in
paediatric patients initiating treatment with ORKAMBI. ORKAMBI has
not been studied in patients with CF who have undergone organ
transplantation. Therefore, use in transplanted patients is not
recommended.
PBS Information: ORKAMBI is not listed on the PBS
About the Australian Funding Process
Australian approval and reimbursement of a new medicine is a
multi-step process. Generally, once a new medicine receives
approval from the TGA, it is assessed for effectiveness and
cost-effectiveness by the Pharmaceutical Benefits Advisory
Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme
(PBS). However, the TGA-PBAC parallel process allows for a
reimbursement submission to be made to the PBAC once the TGA
submission has been made. Additional information regarding the
reimbursement of ORKAMBI in Australia is expected to be available
in April 2016, following the publicly announced planned review of
ORKAMBI at the PBAC meeting, March 9 to 11, 2016.
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 cystic fibrosis
transmembrane conductance regulator (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, or genotyping test, lead to CF by creating
non-working or too few CFTR proteins 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. In 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.
In Australia, cystic fibrosis is the most common, genetically
acquired, life-shortening chronic illness affecting young people.
In 2013, the average age of death due to CF in Australia was 27
years.
One in 2,500 babies in Australia, or one baby every four days,
is born with CF. Approximately one million Australians, or one in
25 people, carry the CF gene, the majority of whom are unaware they
are carriers. Instituted in all Australian States and Territories
in 1986, newborn screening drives early diagnosis: almost 90
percent of infant diagnoses are made before three months of
age.
Chronic antibiotic treatment is widespread among Australians
with CF regardless of age. In 2013, 92 percent of the youngest
patients, those under 2 years, received treatment with antibiotics,
and at least 95 percent of those in all other age groups also
received antibiotic treatment. Nearly half (42.5 percent) of
Australians with CF were hospitalised at least once in 2013. Of
these patients, approximately half (47.1 percent) spent at least 14
days in the hospital.
In Australia, more than half (51 percent) of people with CF have
two copies of the most common CFTR mutation, known as F508del.
Approximately 1,000 people with CF in Australia 12 years and older
have two copies of the F508del mutation.
About ORKAMBI® 200/125 (lumacaftor 200mg and
ivacaftor 125mg) and the F508del mutation
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, a CFTR corrector, 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, a CFTR potentiator, which is
designed to enhance the function of the CFTR protein once it
reaches the cell surface. It is an oral tablet taken every 12 hours
– two tablets in the morning and two tablets in the evening.
For complete product information, please see the Consumer
Medical Information (CMI) and Product Information (PI) that can be
found on www.tga.gov.au once posted.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 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.
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, as
amended, including the quote in the second paragraph of this press
release and statements regarding the funding process in Australia.
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
commercializing ORKAMBI in Australia 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 Vertex'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,
+1-617-341-6108orEric Rojas, +1-617-961-7205orZach Barber
+1-617-341-6470OrMedia:mediainfo@vrtx.comorEurope &
Australia,+44 20 3204 5275orNorth America, +1 617 341 6992
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