-Approximately 8,500 people in the U.S. are
ages 12 and older and have two copies of the F508del mutation, the
most common genetic form of the disease-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) approved
ORKAMBI™ (lumacaftor/ivacaftor), the first medicine to treat the
underlying cause of cystic fibrosis (CF) in people ages 12 and
older with two copies of the F508del mutation. It is only indicated
for these patients, who can be identified with a genetic test.
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Cystic fibrosis is a rare, life-threatening genetic disease.
People with two copies of the F508del mutation represent the
largest group of people with CF. Of the 30,000 people in the United
States with CF, approximately 8,500 ages 12 and older have two
copies of the F508del mutation. ORKAMBI will be available for
shipment to specialty pharmacies in the United States within
days.
“Today is a remarkable day for science, medicine and the CF
community,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s Chairman,
President and Chief Executive Officer. “More than 15 years ago, our
scientists set out to discover and develop medicines to treat the
underlying cause of cystic fibrosis. Today, the approval of ORKAMBI
represents a fundamental change in the treatment of the most common
form of CF, marking significant progress for us and for the entire
CF community. While we celebrate this important step forward, we
also recognize that two out of three patients in the U.S. still do
not have a medicine to treat the underlying cause of their disease.
We share their urgency and are committed to continuing our
significant investment in research and development to discover new
medicines for them and to improve upon what we offer patients
today.”
Vertex will host an investor conference call on Thursday, July
2, at 2:15 p.m. ET. to provide more information on the approval of
ORKAMBI.
The approval of ORKAMBI was based on data from two Phase 3
studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100
people with CF ages 12 and older with two copies of the F508del
mutation. Patients treated with ORKAMBI experienced statistically
significant improvements in lung function. Patients also
experienced reductions in pulmonary exacerbations and improvements
in body mass index (BMI). 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).
Vertex continues to invest in CF research and development with
the goal of treating the vast majority of people with the disease
and enhancing the benefit for those we treat. Multiple Phase 2 and
Phase 3 clinical studies are in progress and Vertex has an ongoing
research program focused on discovering new CF medicines.
“In 1998, Vertex and the CF Foundation embarked on a scientific
challenge that many believed would be impossible – to discover
medicines that treat the cause of CF,” said Robert J. Beall, Ph.D.,
President and CEO of the Cystic Fibrosis Foundation. “Today’s
approval is a milestone for the CF community. We congratulate
Vertex for their success in developing new CF medicines and are
pleased with their continuing commitment to help all eligible
patients get access to these medicines.”
Helping Patients Access ORKAMBI
The people who work at Vertex understand that medicines can
only help patients who can get them. The Vertex Guidance &
Patient Support (Vertex GPS™) program provides a dedicated team of
Vertex employees who help eligible patients who have been
prescribed our medicines within their labeled
indications understand their insurance benefits and the
resources that are available to help them.
Vertex also offers a co-pay assistance program for patients
with commercial insurance coverage and a free medicine program for
qualifying patients who are uninsured and who meet certain income
and other eligibility criteria. More information is available by
visiting www.VertexGPS.com or by calling 1-877-752-5933.
About CF and ORKAMBI
Cystic fibrosis is a rare genetic disease that is caused by
defective or missing cystic fibrosis transmembrane conductance
regulatory (CFTR) proteins resulting from mutations in
the CFTR gene. The defective or missing proteins result
in poor flow of salt and water into or out of the cell in a number
of organs, including the lungs. 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. ORKAMBI is
taken every 12 hours - once in the morning and once in the
evening.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI™
(lumacaftor/ivacaftor) TABLETS
ORKAMBI is a combination of lumacaftor and ivacaftor indicated
for the treatment of cystic fibrosis (CF) in patients age 12 years
and older who are homozygous for the F508del mutation in the CFTR
gene. The efficacy and safety of ORKAMBI have not been established
in patients with CF other than those homozygous for the F508del
mutation.
Worsening of liver function, including hepatic encephalopathy,
in patients with advanced liver disease has been reported in some
patients with CF while receiving ORKAMBI. ORKAMBI should be used
with caution in patients with advanced liver disease and only if
the benefits are expected to outweigh the risks. If ORKAMBI is used
in these patients, the patients should be closely monitored and the
dose reduced.
Serious adverse reactions related to elevated transaminases have
been reported in patients with CF receiving ORKAMBI and, in some
instances, associated with concomitant elevations in total serum
bilirubin. It is recommended that ALT, AST, and bilirubin be
assessed prior to initiating ORKAMBI, 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. Patients who develop increased
ALT, AST, or bilirubin should be closely monitored until the
abnormalities resolve. Dosing should be interrupted in patients
with ALT or AST greater than 5x upper limit of normal (ULN) when
not associated with elevated bilirubin. Dosing should also be
interrupted in patients with ALT or AST elevations greater than 3x
ULN when associated with bilirubin elevations greater than 2x ULN.
Following resolution of transaminase elevations, consider the
benefits and risks of resuming dosing.
Respiratory events (e.g., chest discomfort, shortness of breath,
and chest tightness) were observed more commonly in patients during
initiation of ORKAMBI compared to those who received placebo.
Clinical experience in patients with percent predicted FEV1 <40
is limited, and additional monitoring of these patients is
recommended during initiation of therapy.
Co-administration of ORKAMBI with sensitive CYP3A substrates or
CYP3A substrates with a narrow therapeutic index is not recommended
as ORKAMBI may reduce their effectiveness.
ORKAMBI may substantially decrease hormonal contraceptive
exposure, reducing their effectiveness and increasing the incidence
of menstruation-associated adverse reactions. Hormonal
contraceptives, including oral, injectable, transdermal, and
implantable, should not be relied upon as an effective method of
contraception when co-administered with ORKAMBI.
Co-administration with strong CYP3A inducers (e.g. rifampin,
rifabutin, phenobarbital, carbamazepine, phenytoin and St. John’s
wort) is not recommended as they may reduce the therapeutic
effectiveness of ORKAMBI.
ORKAMBI has the potential to affect other drugs. For additional
information regarding drug interactions, see full Prescribing
Information.
Abnormalities of the eye lens (cataracts) have been reported in
pediatric patients treated with ivacaftor, a component of ORKAMBI.
Baseline and follow-up ophthalmological examinations are
recommended in pediatric patients initiating treatment with
ORKAMBI.
Serious adverse reactions that occurred more frequently in
patients treated with ORKAMBI included pneumonia, blood in sputum,
cough, increased muscle enzyme levels, and liver enzyme elevations.
The most common adverse reactions associated with ORKAMBI include
shortness of breath, sore throat, nausea, diarrhea, upper
respiratory tract infection, fatigue, chest tightness, increased
blood creatinine phosphokinase, rash, flatulence, runny nose, and
influenza.
Please see full prescribing information for ORKAMBI available at
www.ORKAMBI.com.
Global Regulatory Submissions for ORKAMBI
Outside of the U.S., Vertex has submitted ORKAMBI for regulatory
approval in the European Union, Australia and Canada. A decision by
the European Medicines Agency (EMA) is anticipated by the end of
2015. Reviews by Health Canada and Australia’s Therapeutic Goods
Administration (TGA) are also ongoing.
Investor Conference Call
Vertex will host an investor conference call and webcast on
Thursday, July 2, at 2:15 p.m. ET. To listen to the live call on
the telephone dial (866) 501-1537 (United States and Canada) or
(720) 545-0001 (International). The conference ID number for the
live call and replay is 76077705. In addition, the conference call
will be webcast live, and a link to the webcast may be accessed
through Vertex's website at www.vrtx.com in the “Investors"
section under the "Events & Presentations" page.
The call will be available for replay via telephone and webcast.
The replay phone number in the United States and Canada is (855)
859-2056. The international replay number is (404) 537-3406. The
archived webcast will be available at www.vrtx.com.
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 with CF is 41 years, but the median age of
death is 27 years.
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. Both of
our approved CF medicines 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
five 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 statements by Dr. Leiden in the third
paragraph of this press release and statements regarding (i) the
timing of the availability of ORKAMBI for shipment to specialty
pharmacies in the United States; (ii) Vertex’s commitment to
continuing its significant investment in research and development
programs in cystic fibrosis; and (iii) the anticipated timing of
the completion of regulatory reviews in international markets.
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 the United States, obtaining approval
and commercializing ORKAMBI in international markets, developing
additional medicines to treat cystic fibrosis 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)
© 2015 Vertex Pharmaceuticals Incorporated I VRX-US-02-01002 I
07/2015
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version on businesswire.com: http://www.businesswire.com/news/home/20150702005760/en/
Vertex Pharmaceuticals IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orKelly Lewis,
617-961-7530orMedia: mediainfo@vrtx.comUS:
617-341-6992Europe & Australia: +41 22 593 6066
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