- ORKAMBI is the first medicine in Canada to
treat the underlying cause of CF in young children with two copies
of the F508del mutation -
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today
announced that Health Canada has granted Market Authorization for
ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2
through 5 years with cystic fibrosis (CF) who have two copies of
the F508del CFTR mutation.
“We believe it is important to treat the underlying cause of
cystic fibrosis as early as possible and are committed to expanding
treatment options for young children,” said Reshma Kewalramani,
M.D., Executive Vice President and Chief Medical Officer at Vertex.
“This approval, along with the progress we’ve made in our research
and development for multiple potential new medicines over the last
year, is a testament to how far we’ve come on our goal to bring
effective medicines to all people living with CF.”
Approval is based on a Phase 3 open-label safety study in 60
patients that showed treatment with ORKAMBI was generally well
tolerated for 24 weeks, with a safety profile similar to that in
patients ages 6-11 years. Improvements in sweat chloride, a
secondary endpoint, were observed at week 24 (mean decrease in
sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6,
n=49).
In the Phase 3 study, researchers also saw changes in key growth
parameters, which were also secondary endpoints in the study. The
most common adverse event (≥30%) was cough (63%); most adverse
events were mild or moderate in severity. Four patients experienced
serious adverse events (2 pulmonary exacerbations, 1
gastroenteritis, 1 constipation) and three patients discontinued
treatment due to treatment emergent adverse events of elevated
liver function tests. These findings were presented at the 41st
European Cystic Fibrosis Society Conference in June 2018.
ORKAMBI was approved by the U.S Food and Drug Administration
(FDA) in August of this year for use in patients ages 2 to 5 years
who have two copies of the F508del CFTR mutation. A Marketing
Authorization Application (MAA) line extension for ORKAMBI in
children ages 2 through 5 years has been submitted to the European
Medicines Agency (EMA). The European Medicines Agency’s (EMA)
Committee for Medicinal Products for Human Use (CHMP) adopted a
positive opinion for ORKAMBI for use in this patient population in
November, and the company expects a decision from the European
Commission in the coming months.
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 cystic fibrosis
transmembrane conductance regulator (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 proteins
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)
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.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor)
ORKAMBI is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients age 2 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 2 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 and anti-anxiety
medicines triazolam or midazolam; the immunosuppressant medicines
cyclosporine, everolimus, sirolimus, or tacrolimus; or St. John’s
wort.
Before taking ORKAMBI, patients should tell their doctor
about all their medical conditions, including if they: have or
have had liver problems; have kidney problems; have had an organ
transplant; or are using birth control. Hormonal contraceptives,
including oral, injectable, transdermal, or implantable forms
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.
ORKAMBI can cause serious side effects, including:
Worsening of liver function in people with severe liver
disease. The worsening of liver function can be serious or cause
death. Patients should talk to their doctor if they have been told
they have liver disease as their doctor may need to adjust the dose
of ORKAMBI.
High liver enzymes in the blood, which can be a sign of
liver injury. 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.
Breathing problems such as shortness of breath or chest
tightness in patients when starting ORKAMBI, especially in patients
who have poor lung function. If a patient has poor lung function,
their doctor may monitor them more closely when starting
ORKAMBI.
An increase in blood pressure in some people receiving
ORKAMBI. The patient’s doctor should monitor their blood pressure
during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) in some children
and adolescents receiving ORKAMBI. For children and adolescents,
the patient’s doctor should perform eye examinations before and
during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include:
breathing problems, such as shortness of breath and chest
tightness; nausea; diarrhea; fatigue; increase in a certain blood
enzyme called creatinine phosphokinase; rash; gas; common cold,
including sore throat, stuffy or runny nose; flu or flu-like
symptoms; and irregular, missed, or abnormal periods (menses) and
increase in the amount of menstrual bleeding.
Side effects seen in children are similar to those seen
in adults and adolescents. Additional common side effects seen in
children include: cough with sputum, stuffy nose, headache, stomach
pain, and increase in sputum.
Please click here to see the full U.S. Prescribing
Information for ORKAMBI (lumacaftor/ivacaftor).
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 nine 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), SYMDEKO®
(tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445 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, as
amended, including the statements by Dr. Kewalramani in the second
paragraph and statements regarding the anticipated timing of an EMA
decision on the MAA line extension. While the company 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 company's drug candidates 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
Vertex'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,
617-341-6108
Eric Rojas, 617-961-7205
Zach Barber, 617-341-6470
Media:mediainfo@vrtx.com
North America:Christina Cunningham, +1 647-790-1602
Europe & Australia: +44 20 3204 5275
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