- In Europe, there are approximately 3,400
children ages 6-11 who have two copies of the F508del mutation
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Union Committee for Medicinal Products
for Human Use (CHMP) issued a positive opinion recommending
extension of the Marketing Authorization for ORKAMBI®
(lumacafator/ivacaftor) to children with cystic fibrosis (CF) ages
6 through 11 who have two copies of the F508del mutation. There are
approximately 3,400 patients ages 6 through 11 eligible for this
medicine in Europe.
“Cystic fibrosis is a systemic, multi-organ, progressive disease
present from birth,” said David Gillen, M.D., Head of International
Medical Affairs at Vertex. “This recommendation brings us closer to
being able to help more people with CF who currently do not have a
medicine to treat the underlying cause of their disease.”
Positive CHMP opinions are recommendations for approval to the
European Commission, which has the authority to approve medicines
for the European Union. The European Commission generally follows
the recommendations of the CHMP and typically extends the Marketing
Authorization within three months. In some countries, we have in
place existing agreements that will make ORKAMBI available to this
age group immediately following Marketing Authorization. In other
countries, Vertex will begin the country-by-country reimbursement
process following Marketing Authorization.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening 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, 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 protein
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 in many patients that
eventually leads to death. The median age of death is in the
mid-to-late 20s.
About
ORKAMBI® (lumacaftor/ivacaftor)
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. It is an oral
pill taken every 12 hours - once in the morning and once in the
evening.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor)
TABLETSORKAMBI is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 6 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 6 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; 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. Patients should
tell their doctor if they are pregnant or plan to become pregnant
(it is unknown if ORKAMBI will harm the unborn baby) or if they 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, patients 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. Patients
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 ORKAMBI and 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
U.S. Prescribing Information for ORKAMBI.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters
is now located in Boston's Innovation District. Today,
the company has research and development sites and commercial
offices in the United
States, Europe, Canada and Australia. Vertex is
consistently recognized as one of the industry's top places to
work, including being named to Science magazine's Top
Employers in the life sciences ranking for eight years in a row.
For additional information and the latest updates from the company,
please visit www.vrtx.com.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation.
KALYDECO® (ivacaftor), ORKAMBI®(lumacaftor/ivacaftor),
tezacaftor, VX-440, VX-152 and VX-659 were discovered by Vertex as
part of this collaboration.
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. Gillen’s statement in the second
paragraph of this press release and statements regarding the
European Commission generally following the recommendations of the
CHMP and typically issuing marketing approval within three months.
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 could
experience unforeseen delays in obtaining marketing approval from
the European Commission 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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version on businesswire.com: http://www.businesswire.com/news/home/20171110005317/en/
Vertex Pharmaceuticals IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comorNorth America:Megan
Goulart, + 1-617-341-6992orEurope & Australia:Rebecca
Hunt, +44 7718 962 690
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