Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
approved KALYDECO® (ivacaftor) for use in more than 600 people with
cystic fibrosis (CF) ages 2 and older who have one of five residual
function mutations that result in a splicing defect in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene. This
approval was based on Phase 3 clinical data for KALYDECO in these
mutations and follows the FDA’s approval of KALYDECO in May 2017
for 23 other residual function mutations, which was based on
analyses of in vitro data. Both approvals are supported by more
than five years of real-world clinical experience that demonstrate
KALYDECO's established safety and efficacy profile. Based on
today’s approval, Vertex increased its guidance for 2017 KALYDECO
product revenues to a range of $770 million to $800 million.
Vertex’s guidance range for total CF product revenues in 2017 is
now $1.87 billion to $2.1 billion, including ORKAMBI guidance of
$1.1 billion - $1.3 billion.
“In the five years since KALYDECO became the first approved
medicine to treat the underlying cause of cystic fibrosis, we have
been relentless in our efforts to bring this important medicine to
all who may benefit,” said Jeffrey Chodakewitz, M.D., Executive
Vice President and Chief Medical Officer at Vertex. “We will
continue to pursue this goal until all people with CF have a
medicine that treats their form of this serious and life-shortening
disease.”
CF is caused by defective or missing cystic fibrosis
transmembrane conductance regulator (CFTR) proteins resulting from
mutations in the CFTR gene. The defective or missing
proteins result in poor flow of salt (sodium and chloride) and
water into or out of cells in a number of organs, including the
lungs. The five mutations covered under today’s approval
(2789+5G—>A, 3272-26A—>G, 3849+10kbC—>T, 711+3A—>G, and
E831X) cause CF and result in a moderate loss of chloride
transport. People who have these mutations generally experience
progressive lung function decline and other complications of the
disease. All five of these mutations were evaluated as part of the
previously disclosed Phase 3 EXPAND study in which the KALYDECO
monotherapy arm met its primary efficacy endpoint and was generally
well tolerated.
KALYDECO is now approved in the U.S. to treat people with CF
ages 2 and older who have one of 38 ivacaftor-responsive mutations
in the CFTR gene.
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
currently the median age of death is 29 years.
About KALYDECO® (ivacaftor)
KALYDECO (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, KALYDECO is an oral
medicine designed to keep CFTR proteins at the cell surface open
longer to improve the transport of salt and water across the cell
membrane, which helps hydrate and clear mucus from the airways.
KALYDECO is available as 150 mg tablets for adults and pediatric
patients age 6 years and older, and is taken with fat-containing
food. It is also available as 50 mg and 75 mg granules in pediatric
patients ages 2 to less than 6 years and is administered with
soft-food or liquid with fat-containing food.
People with CF who have specific mutations in the CFTR gene are
currently benefiting from KALYDECO in 27 different countries across
North America, Europe and Australia.
KALYDECO® (ivacaftor) INDICATION AND IMPORTANT
SAFETY INFORMATION
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 2 years and older
who have at least one mutation in their CF gene that is responsive
to KALYDECO. Patients should talk to their doctor to learn if they
have an indicated CF gene mutation. 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.
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) was discovered by Vertex as part of this
collaboration.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex’s headquarters
is now located in Boston’s Innovation District. Today, the company
has research and development sites and commercial offices
in the United States, Europe, Canada and
Australia. Vertex is consistently recognized as one of the
industry’s top places to work, including being named
to Science magazine’s Top Employers in the life sciences
ranking for seven years in a row. 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, the statements in the second
paragraph of the press release and statements regarding guidance
for 2017 KALYDECO and ORKAMBI net product revenues. 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 the company's expectations regarding its
2017 KALYDECO and ORKAMBI net product revenues may be incorrect
(including because one or more of the company's assumptions
underlying its expectations may not be realized), 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 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.comorNorth
America:Heather Nichols, +1 617-961-0534orEurope &
Australia:Megan Goulart, +44 20 3204 5275
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