- Precision medicine decision based on in vitro
data and supported by more than five years of real-world clinical
data that demonstrate KALYDECO’s strong safety and efficacy profile
for eligible patients -
- Vertex working with FDA to obtain rapid
approval for more than 600 additional people who have mutations
responsive to KALYDECO, including one of five “splice” mutations
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
approved KALYDECO® (ivacaftor) for use in people with cystic
fibrosis (CF) ages 2 and older who have one of 23 residual function
mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene. This precision medicine decision is based on
analyses of in vitro data and is supported by more than five years
of real-world clinical data that demonstrate KALYDECO’s strong
safety and efficacy profile. More than 900 people ages 2 and older
in the U.S. have one of these mutations. Based on this approval,
Vertex today increased its guidance for 2017 product revenues of
KALYDECO to a range of $740 million to $770 million.
In addition to the mutations added to the label today, Vertex is
continuing discussions with the FDA concerning the approval for
additional people who have mutations responsive to KALYDECO,
including one of five “splice” mutations. These five mutations were
evaluated as part of the previously disclosed Phase 3 EXPAND study
in which the KALYDECO monotherapy arm met its primary efficacy
endpoint while being generally well tolerated. More than 600 people
ages 2 and older in the U.S. have one of these mutations.
“Five years ago, KALYDECO became the first medicine to treat the
underlying cause of CF. Since then, we have continued to invest in
studies to improve the understanding of how this important medicine
may benefit others with this serious and life-shortening disease,”
said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief
Medical Officer at Vertex. “We are encouraged by the FDA’s
willingness to explore innovative ways to make highly effective
medicines like KALYDECO with a well-established safety profile
available to more people who are in urgent need. We will continue
to work closely with the FDA to bring KALYDECO to more people with
responsive mutations who are still in need as rapidly as
possible.”
“CF treatment has advanced rapidly, but there is need for
broader access to these important medicines and development of
additional medicines remains urgent,” said Patrick Flume, M.D.,
Director of the Medical University of South Carolina Cystic
Fibrosis Center. “The use of in vitro data to support this approval
is an important step forward in making medicines like KALYDECO
available to more patients, especially those with rare
mutations.”
Based on clinical data from numerous prior studies and a
demonstrated in vitro response employing a well-established,
analytically validated method, KALYDECO is now approved to treat
people with CF ages 2 and older who have one of 33 mutations in the
CFTR gene.
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 (chloride) and water into and
out of the cell in a number of organs, including the lungs.
Chloride transport is a marker of the function of the CFTR protein
at the cell surface. As with the other mutations for which KALYDECO
is approved, mutations covered under today’s approval are known to
have some CFTR protein at the cell surface and have shown in vitro
increases in chloride transport in response to KALYDECO by at least
10 percent of normal over baseline. Similar to the R117H mutation
for which KALYDECO was previously approved, these additional
residual function mutations result in a moderate loss of CFTR
chloride transport, and people with these mutations generally have
progressive lung function decline and other complications of
CF.
Pipeline Progress for Residual Function Mutations
On March 28, 2017, Vertex announced positive results from a
Phase 3 study that evaluated tezacaftor/ivacaftor combination
treatment in people who have one mutation that results in residual
CFTR function and one F508del mutation. The study met its primary
endpoint and the combination was generally well tolerated. Vertex
plans to submit these data as part of a New Drug Application (NDA)
to the U.S. Food and Drug Administration (FDA) and a Marketing
Authorization Application (MAA) to the European Medicines Agency
(EMA) for the tezacaftor/ivacaftor combination early in the third
quarter of 2017.
About Cystic Fibrosis and
KALYDECO® (ivacaftor)
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.
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. In
adults and pediatric patients age 6 years and older one 150 mg
tablet is taken orally every 12 hours with fat-containing food.
Pediatric patients ages 2 to less than 6 years who weigh less than
14 kg take one 50 mg packet of oral granules mixed with 1 teaspoon
(5 mL) of soft food or liquid administered orally every 12 hours
with fat-containing food. Pediatric patients ages 2 to less than 6
years of age who weigh 14 kg or greater take one 75 mg packet of
oral granules with 1 teaspoon (5 mL) of soft food or liquid
administered orally every 12 hours 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 the 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 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
seven 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, the statements in the third and
fourth paragraphs of the press release and statements regarding (i)
the potential approval of KALYDECO for patients with “splice”
mutations, (ii) Vertex’s updated guidance for KALYDECO 2017 net
product revenues and (iii) Vertex’s plans to submit an NDA and MAA
for tezacaftor/ivacaftor. 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 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:David Whitrap, +1 617-961-5093orEurope &
Australia:Megan Goulart, +44 20 3204 5275
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