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- In Europe, there are approximately 3,400 children ages 6-11 who have two
copies of the F508del mutation -
BOSTON --(BUSINESS WIRE)--10.11.2017--
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^(R) (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
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^(R) (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^(R) (lumacaftor/
ORKAMBI 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
Please click here to see the full U.S. Prescribing Information for ORKAMBI.
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^(R) (ivacaftor), ORKAMBI^(R)(lumacaftor/ivacaftor),
tezacaftor, VX-440, VX-152 and VX-659 were discovered by Vertex as part of this
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.
View source version on businesswire.com: http://www.businesswire.com/news/home/
Vertex Pharmaceuticals Incorporated
Michael Partridge, 617-341-6108
Eric Rojas, 617-961-7205
Zach Barber, 617-341-6470
Megan Goulart, + 1-617-341-6992
Europe & Australia:
Rebecca Hunt, +44 7718 962 690
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