-Opportunity to treat the underlying cause of
CF earlier than ever before-
-Safety data from Phase 3 ARRIVAL study support
treatment with KALYDECO in children ages six to <12 months with
eligible mutations-
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today
announced the U.S. Food and Drug Administration (FDA) approved
KALYDECO® (ivacaftor) for use in children with cystic fibrosis (CF)
ages six months to less than 12 months who have at least one
mutation in their cystic fibrosis transmembrane conductance
regulator (CFTR) gene that is responsive to KALYDECO based on
clinical and/or in vitro assay data. KALYDECO is already approved
in the U.S., Canada and EU for the treatment of CF in patients ages
12 months and older.
“Today’s approval for KALYDECO allows physicians to begin
treating the underlying cause of CF in eligible infants as young as
six months of age for the first time, with the potential to modify
the course of the disease,” said Margaret Rosenfeld, M.D., MPH,
Seattle Children’s Research Institute and Department of Pediatrics,
University of Washington School of Medicine.
This FDA approval is based on data from a 24-week Phase 3
open-label safety cohort (ARRIVAL) of 11 children with CF aged six
months to less than 12 months who have one of 10 mutations in the
CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N,
S1255P, G1349D or R117H). The study demonstrated a safety profile
similar to that observed in previous Phase 3 studies of older
children and adults; most adverse events were mild or moderate in
severity, and no patient discontinued therapy due to adverse
events. The most common adverse events (≥30%) were cough (64%),
nasal congestion (36%) and rhinorrhea (36%). Three serious adverse
events, all considered unrelated to study treatment by the
investigators, were observed in three patients.
Mean baseline sweat chloride for the children in this cohort was
101.5 mmol/L (n=11). Following 24 weeks of treatment with KALYDECO,
the mean sweat chloride level was 43.1 mmol/L (n=6). In the six
subjects with paired sweat chloride samples at baseline and week
24, there was a mean absolute change of -58.6 mmol/L (95% CI;
-75.9, -41.3).
Results of this study were presented at the 32nd Annual North
American Cystic Fibrosis Conference in October 2018.
“The manifestations of CF are often present at birth, which
underscores our relentless commitment to reach the youngest CF
patients possible in our clinical trials,” said Reshma Kewalramani,
M.D., Executive Vice President and Chief Medical Officer at Vertex.
“As an important outcome of these efforts, we are now able to treat
infants with cystic fibrosis as early as six months of age with
KALYDECO.”
KALYDECO was first approved in 2012 in the U.S. and is now
available in more than 40 countries with more than 5,000 patients
on therapy. For more information on KALYDECO, prescribing
information, or patient assistance programs, visit Kalydeco.com or
VertexGPS.com.
About Cystic FibrosisCystic Fibrosis (CF) 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 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 KALYDECO® (ivacaftor)KALYDECO® (ivacaftor) is the
first medicine to treat the underlying cause of CF in people with
specific mutations in the CFTR gene. Known as a CFTR potentiator,
KALYDECO is an oral medicine designed to keep CFTR proteins at the
cell surface open longer to improve the transport of salt and water
across the cell membrane, which helps hydrate and clear mucus from
the airways. KALYDECO is available as 150 mg tablets for adults and
pediatric patients age 6 years and older. It is also available as
25 mg, 50 mg and 75 mg granules in pediatric patients ages 6 months
to less than 6 years.
People with CF who have specific mutations in the CFTR gene are
currently indicated for KALYDECO in different countries across
North America, Europe and other International markets.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO®
(ivacaftor)KALYDECO (ivacaftor) is a prescription medicine used
for the treatment of cystic fibrosis (CF) in patients age 6 months
and older who have at least one mutation in their CF gene that is
responsive to KALYDECO. Patients should talk to their doctor to
learn if they have an indicated CF gene mutation. It is not known
if KALYDECO is safe and effective in children under 6 months of
age.
Patients should not take KALYDECO if they take certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; seizure medications such as phenobarbital,
carbamazepine, or phenytoin; or St. John’s wort.
Before taking KALYDECO, patients should tell their doctor if
they: have liver or kidney problems; drink grapefruit juice, or
eat grapefruit or Seville oranges; are pregnant or plan to become
pregnant because it is not known if KALYDECO will harm an unborn
baby; and are breastfeeding or planning to breastfeed because is
not known if KALYDECO passes into breast milk.
KALYDECO may affect the way other medicines work, and other
medicines may affect how KALYDECO works. Therefore the dose of
KALYDECO may need to be adjusted when taken with certain
medications. Patients should especially tell their doctor if they
take antifungal medications such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
needs them to be alert until they know how KALYDECO affects
them.
Patients should avoid food containing grapefruit or
Seville oranges while taking KALYDECO.
KALYDECO can cause serious side effects.
High liver enzymes in the blood have been reported in
patients receiving KALYDECO. The patient’s doctor will do blood
tests to check their liver before starting KALYDECO, every 3 months
during the first year of taking KALYDECO, and every year while
taking KALYDECO. For patients who have had high liver enzymes in
the past, the doctor may do blood tests to check the liver more
often. Patients should call their 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 their
skin or the white part of their eyes; loss of appetite; nausea or
vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in
some children and adolescents receiving KALYDECO. The patient’s
doctor should perform eye examinations prior to and during
treatment with KALYDECO to look for cataracts.
The most common side effects include headache; upper
respiratory tract infection (common cold), which includes sore
throat, nasal or sinus congestion, and runny nose; stomach
(abdominal) pain; diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing
Information for KALYDECO.
About VertexVertex 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, Australia and Latin America. 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 StatementsThis
press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including,
without limitation, the statements in the second and sixth
paragraphs of the press release. 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 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:mediainfo@vrtx.comorNorth America: +
1-617-341-6992orEurope & Australia: + 44 20 3204 5275
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