-Approximately 2,400 children ages 6
to 11 have two copies of the F508del mutation in the U.S.-
-Target review date of September 30, 2016 set
for the FDA’s decision on the application-
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 ORKAMBI® (lumacaftor/ivacaftor) in people with cystic
fibrosis (CF) ages 6 to 11 who have two copies of the F508del
mutation. The FDA granted Vertex's request for Priority Review of
this sNDA, and a target review date of September 30, 2016 was set
under the Prescription Drug User Fee Act (PDUFA).
“As complications related to CF can occur early in life, we
believe it is important to begin treatment as early as possible and
this supplemental New Drug Application for approval of ORKAMBI in
children as young as six is an important step in that direction,”
said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief
Medical Officer at Vertex. “This submission demonstrates Vertex’s
continued progress toward our goal of developing medicines for all
people with CF.”
The sNDA is based on data from an open label Phase 3 clinical
safety study of ORKAMBI. Data from this study will be presented at
the 39th European Cystic Fibrosis Society conference being held
June 8 to 11 in Basel, Switzerland.
To support potential approval in the European Union, a six-month
Phase 3 efficacy study of children ages 6 to 11 is ongoing. Vertex
recently completed enrollment in this study and, pending data from
the study, plans to submit a Marketing Authorization Application
(MAA) variation in the European Union in the first half of 2017 for
children ages 6 to 11 who have two copies of the F508del mutation.
The primary endpoint of this efficacy study is the absolute change
in lung clearance index (LCI). There are approximately 3,400
children ages 6 to 11 who have two copies of the F508del mutation
in the European Union.
About CF and ORKAMBI
Cystic fibrosis is a rare genetic disease that 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 and water into or out of the cell in a number
of organs, including the lungs. In people with two copies of the
F508del mutation, the CFTR protein is not processed and trafficked
normally within the cell, resulting in little to no CFTR protein at
the cell surface. Patients with two copies of the F508del mutation
are easily identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to
increase the amount of mature protein at the cell surface by
targeting the processing and trafficking defect of the F508del CFTR
protein, and ivacaftor, which is designed to enhance the function
of the CFTR protein once it reaches the cell surface. ORKAMBI is
taken every 12 hours - once in the morning and once in the evening
with fat-containing food.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients age 12 years and older who have
two copies of the F508del mutation (F508del/F508del) in their CFTR
gene. ORKAMBI should only be used in these patients. It is not
known if ORKAMBI is safe and effective in children under 12 years
of age.
Patients should not take ORKAMBI if they are taking certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; the seizure medicines phenobarbital,
carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines
triazolam or midazolam; the immunosuppressant medicines everolimus,
sirolimus, or tacrolimus; or St. John’s wort.
Before taking ORKAMBI, patients should tell their doctor if
they have or have had liver problems or have kidney problems;
have had an organ transplant; are using birth control (hormonal
contraceptives, including oral, injectable, transdermal or
implantable forms). Hormonal contraceptives should not be used as a
method of birth control when taking ORKAMBI; are pregnant or plan
to become pregnant because it is unknown if ORKAMBI will harm the
unborn baby; are breastfeeding or planning to breastfeed. It is
unknown if ORKAMBI passes into breast milk.
ORKAMBI may affect the way other medicines work and other
medicines may affect how ORKAMBI works. Therefore, the dose of
ORKAMBI or other medicines may need to be adjusted when taken
together. Patients should especially tell their doctor if they
take: antifungal medicines such as ketoconazole, itraconazole,
posaconazole or voriconazole; or antibiotics such as telithromycin,
clarithromycin, or erythromycin.
When taking ORKAMBI, a patient should tell their doctor
if they stop ORKAMBI for more than 1 week as the doctor may need to
change the dose of ORKAMBI or other medicines the patient is
taking. It is unknown if ORKAMBI causes dizziness. A patient should
not drive a car, use machinery, or do anything requiring alertness
until the patient knows how ORKAMBI affects them.
ORKAMBI can cause serious side effects including:
High liver enzymes in the blood, which can be a sign of liver
injury, have been reported in patients receiving ORKAMBI. The
patient’s doctor will do blood tests to check their liver before
they start ORKAMBI, every three months during the first year of
taking ORKAMBI, and annually thereafter. The patient should call
the 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 the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine; or confusion.
Respiratory events such as shortness of breath or chest
tightness were observed in patients when starting ORKAMBI. If a
patient has poor lung function, their doctor may monitor them more
closely when starting ORKAMBI.
An increase in blood pressure has been seen in some patients
treated with ORKAMBI. The patient’s doctor should monitor their
blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving ivacaftor, a component of
ORKAMBI. For children and adolescents, the patient’s doctor
should perform eye examinations prior to and during treatment with
ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of
breath and/or chest tightness; upper respiratory tract infection
(common cold), including sore throat, stuffy or runny nose;
gastrointestinal symptoms including nausea, diarrhea, or gas; rash;
fatigue; flu or flu-like symptoms; increase in muscle enzyme
levels; and irregular, missed, or abnormal menstrual periods and
heavier bleeding.
Please click here to see the full Prescribing Information for
ORKAMBI.
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 in the United States is
39 years, but the median age of death is 29 years.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation. KALYDECO
(ivacaftor) 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, the target review date of
September 30, 2016 and the expected timing for submission of an MAA
variation in Europe for children ages 6 to 11 who have two copies
of the F508del mutation. 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 data from the Phase 3 safety
study may not be sufficient to support approval of the sNDA, 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:US: 617-341-6992Europe & Australia:
+44 20 3204 5275mediainfo@vrtx.com
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