-Approval provides opportunity to treat the
underlying cause of CF earlier than ever before-
-Safety data from a cohort of the Phase 3
ARRIVAL study support treatment with KALYDECO in children ages four
to <6 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 four months to less than six months old 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. and EU for the treatment of CF in patients ages six
months and older.
“Since the initial approval of KALYDECO more than eight years
ago, we have continued to advance our clinical development program
with the goal of treating the underlying cause of cystic fibrosis
as early in life as possible,” said Reshma Kewalramani, M.D., Chief
Executive Officer and President, Vertex. “Today’s approval is a
testament to our relentless efforts, alongside the clinical and
scientific community, to reach all people with CF who may benefit
from our medicines.”
This FDA approval is based on data from a cohort in the 24-week
Phase 3 open-label safety cohort (ARRIVAL) consisting of 6 children
with CF ages four months to less than six months who have one of 10
mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S,
G1244E, S1251N, S1255P, G1349D or R117H). This cohort demonstrated
a safety profile similar to that observed in older children and
adults.
“Initiating therapy that treats the underlying cause of cystic
fibrosis as early as four months of age may have 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.
KALYDECO was first approved in 2012 in the U.S. and is now
available in more than 40 countries. For more information on
KALYDECO, prescribing information or patient assistance programs,
visit Kalydeco.com or VertexGPS.com.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting approximately 75,000 people worldwide. CF is a
progressive, multi-system disease that affects the lungs, liver, GI
tract, sinuses, sweat glands, pancreas and reproductive tract. CF
is caused by a defective and/or missing CFTR protein resulting from
certain mutations in the CFTR gene. Children must inherit two
defective CFTR genes — one from each parent — to have CF. While
there are many different types of CFTR mutations that can cause the
disease, the vast majority of all people with CF have at least one
F508del mutation. These mutations, which can be determined by a
genetic test, or genotyping test, lead to CF by creating
non-working and/or too few CFTR proteins at the cell surface. The
defective function and/or absence of CFTR protein results in poor
flow of salt and water into and out of the cells 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 early 30s.
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 4 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 4 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; 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 it 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 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 Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
pipeline of investigational small molecule medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney diseases. In addition, Vertex has a rapidly
expanding pipeline of genetic and cell therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is in London, UK. Additionally, the
company has research and development sites and commercial offices
in North America, Europe, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including 10 consecutive years on Science magazine's Top
Employers list and top five on the 2019 Best Employers for
Diversity list by Forbes. For company updates and to learn more
about Vertex's history of innovation, visit www.vrtx.com or follow
us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
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 quotes by Dr. Kewalramani and
Dr. Rosenfeld. 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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