-More than 1,500 people with CF are ages two
and older and have one of these 23 residual function mutations in
the U.S.-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
accepted for review a supplemental New Drug Application (sNDA) for
the use of KALYDECO® (ivacaftor) in people with cystic fibrosis
(CF) ages 2 and older who have one of 23 residual function
mutations. The FDA granted Vertex’s request for Priority Review of
this sNDA, and a target review date of February 6, 2016 was set
under the Prescription Drug User Fee Act (PDUFA) for the FDA's
decision on the sNDA. The submission was based on preclinical and
clinical data showing the effect of ivacaftor on CFTR function in
certain residual function mutations.
“Given the severity of cystic fibrosis, we are committed to
getting KALYDECO to more people as quickly as possible,” said
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief
Medical Officer at Vertex. “Based on the established safety profile
of KALYDECO and our increasing understanding of the biology of
these specific residual function mutations and their response to
ivacaftor, we believe that people with these mutations would
benefit from treatment with this medicine.”
The sNDA was based on preclinical data for ivacaftor in the 23
residual function mutations, the established clinical profile of
KALYDECO and on previously reported data from an exploratory Phase
2a study in 24 people with residual function mutations. In 19 of
the 24 patients enrolled in this study, 8 of the 23 mutations
proposed in the sNDA were represented.
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. KALYDECO is currently approved to treat people
with CF ages 2 and older who have one of 10 mutations in the CFTR
gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N,
S549R or R117H). As with the mutations for which KALYDECO is
currently approved, the 23 residual function mutations in the sNDA
are known to have some CFTR protein at the cell surface and have
shown in vitro increases in chloride transport in response to
ivacaftor in cells expressing the CFTR form produced by each
mutation, characteristics associated with clinical response to
KALYDECO. Similar to the R117H mutation for which KALYDECO was
previously approved, these 23 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.
There are more than 1,500 people ages 2 and older with CF in the
United States who have one of the 23 residual function mutations
included in the sNDA. The 23 residual function mutations included
in the sNDA are: 2789+5G->A, 3849+10kbC->T, 3272-26A->G,
711+3A->G, E56K, P67L, R74W, D110E, D110H, R117C, L206W, R347H,
R352Q, A455E, D579G, E831X, S945L, S977F, F1052V, R1070W, F1074L,
D1152H, and D1270N.
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 one of the following mutations in their CF gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or
R117H.
KALYDECO is not for use in people with CF due to other mutations
in the CF gene. KALYDECO is not effective in patients with CF with
two copies of the F508del mutation (F508del/F508del) in the CF
gene. It is not known if KALYDECO is safe and effective in children
under 2 years of age.
IMPORTANT SAFETY INFORMATION
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. A patient 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. 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.
Please click here to see the full Prescribing Information for
KALYDECO (ivacaftor).
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.
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.
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 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
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 and the target review date of
February 6, 2016. While Vertex 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, that regulatory authorities may not approve, or approve on
a timely basis, the sNDA, that the preclinical data, the
established clinical profile and the data from the exploratory
Phase 2a study may not be sufficient to support approval, 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 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 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, 617-961-7205orKelly Lewis,
617-961-7530orMedia:Zach Barber,
617-341-6992mediainfo@vrtx.com
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