-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.-
Company Website:
http://www.vrtx.com
BOSTON -- (Business Wire)
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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Contacts:
Vertex Pharmaceuticals Incorporated
Investors:
Michael
Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Kelly
Lewis, 617-961-7530
or
Media:
Zach Barber,
617-341-6992
mediainfo@vrtx.com
Source: Vertex Pharmaceuticals Incorporated
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