-Approximately 2,400 children ages 6 through 11
have two copies of the F508del mutation in the U.S.-
- Vertex revises ORKAMBI revenue guidance for
2016 -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) approved
ORKAMBI® (lumacaftor/ivacaftor) for use in children with cystic
fibrosis (CF) ages 6 through 11 who have two copies of the F508del
mutation. People with this mutation represent the largest
population of those with CF, a rare, life-threatening disease.
ORKAMBI is the first and only medicine to treat the underlying
cause of CF for people with this mutation. It was previously
approved by the FDA for use in people ages 12 and older with two
copies of the F508del mutation. With today’s approval,
approximately 11,000 people with CF are eligible for treatment with
ORKAMBI in the United States. ORKAMBI will be available for
eligible children ages 6 through 11 in the United States as soon as
possible. Vertex also today lowered its guidance for 2016 ORKAMBI
revenues to a range of $950 million to $990 million.
“The ability to treat children as young as six who have the most
common form of the disease is an important milestone as we pursue
our goal to develop medicines for all people with CF,” said Jeffrey
Chodakewitz, M.D., Executive Vice President and Chief Medical
Officer at Vertex. “We believe it is important to treat the
underlying cause of the disease as early as possible in these
patients.”
The approval is based on data from a previously announced
open-label Phase 3 clinical safety study of ORKAMBI presented at
the 39th European Cystic Fibrosis Society Conference in June 2016.
These data will be presented at the 30th Annual North American
Cystic Fibrosis Conference October 27-29 in Orlando, Florida.
Vertex plans to submit a Marketing Authorization Application
(MAA) variation in the European Union in the first half of 2017 for
children ages 6 through 11 who have two copies of the F508del
mutation. This application will be based on data from a Phase 3
efficacy study with a primary endpoint of absolute change in lung
clearance index (LCI). These data are expected before the end of
2016.
ORKAMBI Financial Guidance
Vertex today revised its guidance for 2016 ORKAMBI
revenues. The company now expects ORKAMBI revenues of $950
million to $990 million. Vertex’s prior guidance was for total
2016 ORKAMBI revenues of $1.0 billion to $1.1 billion, and to date
Vertex has reported ORKAMBI revenues of $223 million and $245
million for the first and second quarters of 2016,
respectively. Vertex expects third quarter ORKAMBI revenues to
be between $230 million and $235 million. The revised guidance
primarily reflects the following:
- The slower than anticipated launch in
Germany where fewer than 20 percent of the approximately 2,500
eligible patients have initiated treatment to date;
- That we are approaching peak
penetration for ORKAMBI in the U.S. where there are approximately
8,500 eligible patients ages 12 and older; and
- Slower than expected refills for
ORKAMBI during the summer months of July and August.
Today’s approval in people ages 6 through 11 will drive growth
in the U.S. in the fourth quarter of 2016. Growth for ORKAMBI in
2017 will be driven both by obtaining reimbursement approvals in
key European and other countries and by continued growth among
eligible patients ages 6 through 11 in the U.S.
About Cystic Fibrosis and ORKAMBI
Cystic fibrosis is a rare, life-threatening genetic disease
affecting approximately 75,000 people in North
America, Europe and Australia.
CF is caused by defective or missing cystic fibrosis conductance
regulator (CFTR) proteins 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.
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. In pediatric
patients ages 6 through 11, two ORKAMBI tablets (each containing
lumacaftor 100mg/ivacaftor 125mg) are taken orally 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 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 Prescribing
Information for ORKAMBI.
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. ORKAMBI
(lumacaftor/ivacaftor) was 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, the information provided in Dr.
Chodakewitz’s quote in the second paragraph of this press release
and in the section captioned “ORKAMBI Financial Guidance” and
statements regarding (i) Vertex’s revenue guidance and expectations
and (ii) plans to submit an MAA variation in the European Union for
children ages 6 to 11 who have two copies of the F508del mutation
and the timing of expected data from Vertex’s Phase 3 efficacy
study in this patient population. While Vertex believes the
forward-looking statements contained in this press release are
accurate, these forward-looking statements represent the company's
beliefs only as of the date of this press release and 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 the company's expectations regarding revenues may be
incorrect (including because one or more of the company's
assumptions underlying its expectations may not be realized), 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 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:North America:Chris Stamm, +1
617-341-6992mediainfo@vrtx.comorEurope & Australia:Megan
Goulart, +44 20 3204 5275mediainfo@vrtx.com
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