Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA)
approved KALYDECO® for use in children ages 2 to 5 with cystic
fibrosis (CF) who have one of 10 mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene (G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and
R117H). Prior to today’s approval, KALYDECO was approved in the
United States for people ages 6 and older with these mutations.
There are approximately 300 children in the United States ages 2 to
5 who have one of these 10 mutations, including 150 who have the
R117H mutation and 150 who have one of the other nine mutations
that result in a gating defect in the CFTR protein. A new
weight-based oral granule formulation of KALYDECO (50 mg and 75 mg)
that can be mixed in soft foods or liquids was created to meet the
needs of children in this age group who may be unable to swallow a
tablet. The approval is based on previously announced results of an
open-label Phase 3 24-week study that was designed to evaluate the
safety and pharmacokinetics of weight-based dosing of ivacaftor (50
mg or 75 mg twice daily) in children ages 2 to 5. With today’s
approval, more than 3,400 people are currently eligible for
treatment with KALYDECO in the United States, Canada, Europe and
Australia. Cystic fibrosis is caused by a defective or missing CFTR
protein resulting from mutations in the CFTR gene.
“Children with cystic fibrosis can begin to experience
meaningful lung function decline and struggle to gain weight at a
very young age, underscoring the importance of starting treatment
early in life,” said Jeffrey Chodakewitz, M.D., Executive Vice
President and Chief Medical Officer at Vertex. “With today’s
approval, children as young as two years of age now have a medicine
to treat the underlying cause of their CF, bringing us one step
closer to our goal of helping the vast majority of people with this
devastating disease.”
In Europe, an MAA line extension for ivacaftor in children ages
2 to 5 with specific mutations in the CFTR gene has been validated
by the European Medicines Agency (EMA) and is currently
under review by the Committee for Medicinal Products for Human
Use (CHMP).
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
KALYDECO® (ivacaftor)
Ivacaftor is a cystic fibrosis transmembrane conductance
regulatory (CFTR) potentiator indicated for the treatment of cystic
fibrosis (CF). In the U.S. (in patients age 2 years and older) and
Europe (in patients age 6 years and older), ivacaftor is indicated
for patients who have one of the following mutations in the
CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N,
S1255P, S549N, or S549R. In Canada (in patients 6 years and
older), ivacaftor is indicated for patients with these same
mutations and also for patients with the G970R mutation.
Additionally, in the U.S. (in patients age 2 years and older) and
Canada (in patients age 18 years and older) ivacaftor is indicated
for the treatment of CF in patients who have an R117H mutation in
the CFTR gene.
Ivacaftor is available as 150 mg tablets in countries where it
is approved for patients age 6 years and older, and additionally in
the U.S. as 50 mg and 75 mg oral granules for patients age 2 to
less than 6 years.
Ivacaftor is not effective in patients with CF with 2 copies of
the F508del mutation (F508del/F508del) in
the CFTR gene. The safety and efficacy of ivacaftor in
children with CF younger than 2 years of age have not been studied.
The use of ivacaftor in children under the age of 2 years is not
recommended.
High liver enzymes (transaminases; ALT and AST) have been
reported in patients with CF receiving ivacaftor. Transaminase
elevations were more common in patients with a history of
transaminase elevations or in patients who had abnormal
transaminases at baseline. It is recommended that ALT and AST be
assessed prior to initiating ivacaftor, every 3 months during the
first year of treatment, and annually thereafter. For patients with
a history of transaminase elevations, more frequent monitoring of
liver function tests should be considered. Patients who develop
increased transaminase levels should be closely monitored until the
abnormalities resolve. Dosing should be interrupted in patients
with ALT or AST of greater than 5 times the upper limit of normal.
Following resolution of transaminase elevations, consider the
benefits and risks of resuming ivacaftor dosing.
Use of ivacaftor with medicines that are strong CYP3A inducers,
such as the antibiotics rifampin and rifabutin; seizure medications
(phenobarbital, carbamazepine, or phenytoin); and the herbal
supplement St. John's wort, substantially decreases exposure of
ivacaftor and may diminish effectiveness. Therefore,
co-administration is not recommended. The dose of ivacaftor must be
adjusted when used concomitantly with strong and moderate CYP3A
inhibitors or when used in patients with moderate or severe hepatic
disease.
Cases of non-congenital lens opacities/cataracts have been
reported in pediatric patients treated with ivacaftor. Baseline and
follow-up ophthalmological examinations are recommended in
pediatric patients initiating ivacaftor treatment.
Serious adverse reactions that occurred more frequently with
ivacaftor included abdominal pain, increased liver enzymes, and low
blood sugar (hypoglycemia). The most common side effects associated
with ivacaftor include headache; upper respiratory tract infection
(common cold), including 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
ivacaftor. A list of the adverse reactions can be found in the
product labeling for each country where ivacaftor is approved.
Patients should tell their healthcare providers about any side
effect that bothers them or does not go away.
Please see KALYDECO (ivacaftor) U.S. Prescribing
Information, EU Summary of Product
Characteristics, Canadian Product Monograph, Australian
Consumer Medicine Information and Product
Information, Swiss Prescribing Information and Patient
Information, and the New Zealand
Datasheet and Consumer Medicine Information.
About KALYDECO® (ivacaftor)
KALYDECO (ivacaftor) is the first medicine to treat the
underlying cause of CF in people with specific mutations in the
cystic fibrosis transmembrane conductance regulator (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 approved in the
U.S., Europe, Canada, Australia and New
Zealand to treat people with CF who have specific genetic
mutations in the CFTR gene.
Vertex retains worldwide rights to develop and commercialize
KALYDECO.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening 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. In 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
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. This
collaboration was expanded to support the accelerated discovery and
development of Vertex's CFTR modulators.
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,
including, without limitation, Dr. Chodakewitz's statements in the
second paragraph of the press release. 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 regulatory authorities may not approve, or approve on
a timely basis, the company's drug candidates 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)
Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
617-341-6108orKelly Lewis, 617-961-7530orEric Rojas,
617-961-7205orMedia:Zach Barber, 617-767-9533mediainfo@vrtx.com
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