Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that it received a Complete Response Letter from the U.S.
Food and Drug Administration (FDA) for its 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 in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. The FDA determined that it
cannot approve the application in its present form. Vertex
plans to meet with the FDA to determine an appropriate path
forward.
“Our intention with this submission was to rapidly bring
KALYDECO to additional people with CF who we believe may benefit,”
said Vertex Executive Vice President and Chief Medical Officer,
Jeffrey Chodakewitz, M.D. “We chose to pursue this approach given
our strong belief in the science of CF and in the well-established
safety of KALYDECO across many different groups of people with CF.
We are disappointed by this decision and look forward to discussing
with the FDA the next steps to bring KALYDECO to people with CF who
have these residual function mutations.”
The sNDA is based on preclinical data for ivacaftor in residual
function mutations, the established clinical profile of KALYDECO
and on previously reported data from an exploratory Phase 2a study.
In 19 of the 24 patients enrolled in this study, eight 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, which received the FDA’s
Breakthrough Therapy Designation in 2013, is currently approved in
the U.S. 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 approved, this group of 23 residual function
mutations is known to have some functional CFTR protein at the cell
surface. This submission was also based on observed in vitro
increases in chloride transport in response to ivacaftor in cells
expressing CFTR. The presence of CFTR protein at the cell surface
and increases in chloride transport are characteristics associated
with clinical response to KALYDECO.
There are more than 1,500 people ages two 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 six
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 Vertex’s plans with
respect to a potential meeting with the FDA. 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 Vertex may not be able to obtain approval
for KALYDECO as a treatment for people with CF who have residual
function mutations, 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, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comUS:
617-341-6992Europe & Australia: +44 20 3204 5275
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