- More than 600 people with certain rare CF
mutations could become newly eligible for TRIKAFTA, SYMDEKO or
KALYDECO -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the U.S. Food and Drug Administration (FDA) accepted
three supplemental New Drug Applications (sNDAs) for TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO®
(tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).
These regulatory submissions are intended to expand the labels for
TRIKAFTA, SYMDEKO and KALYDECO to include additional rare CFTR
mutations, allowing people with cystic fibrosis (CF) not previously
eligible for these medicines an opportunity to benefit from
treatment that targets the underlying cause of their disease. In
addition, these regulatory submissions may also allow certain
people with CF who are currently eligible for KALYDECO to become
eligible for SYMDEKO or TRIKAFTA and certain people currently
eligible for SYMDEKO may become eligible for TRIKAFTA. The FDA has
assigned a Prescription Drug User Fee Act (PDUFA) target action
date of December 30, 2020. The regulatory submissions are based on
data from an in vitro cell assay showing that these rare CFTR
mutations respond to one or more of these CFTR modulator
regimens.
“We have spent the last 20 years discovering, developing and
bringing new medicines to thousands of people with CF, and the
regulatory submissions announced today are an important next step
in our commitment to bring transformative medicines to everyone
living with this disease,” said David Altshuler, M.D., Ph.D.,
Executive Vice President, Global Research and Chief Scientific
Officer. “Using our well-established in vitro approach, we have
been able to generate data providing evidence that people with
certain rare mutations could benefit from treating the underlying
cause of their disease with CFTR modulators.”
These sNDAs are based on in vitro data from a validated cell
assay model showing that many rare mutations in the CFTR gene are
responsive to one or more of Vertex’s medicines — KALYDECO, SYMDEKO
and TRIKAFTA — beyond the mutations that are currently indicated
for these therapies. Approximately 600 people in the U.S. who have
certain rare CF mutations may benefit from TRIKAFTA, SYMDEKO or
KALYDECO for the first time. In addition, more than 1,100 people
with CF in the U.S. currently eligible for SYMDEKO or KALYDECO may
have the option of an additional CFTR modulator. These regulatory
submissions may allow certain people with CF who are currently
eligible for KALYDECO to become eligible for SYMDEKO or TRIKAFTA
and certain people currently eligible for SYMDEKO may become
eligible for TRIKAFTA.
Data generated from this model, along with Phase 3 clinical
data, have already led to the inclusion of nearly 30 additional
ultra-rare and rare mutations in the U.S. for KALYDECO and SYMDEKO,
including the first ever FDA approval based on in vitro data for a
KALYDECO label expansion in patients with residual function CFTR
mutations.
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), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), and
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
What is KALYDECO?
KALYDECO 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.
What is SYMDEKO?
SYMDEKO 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, or who have at least one mutation
in the CF gene that is responsive to treatment with SYMDEKO.
Patients should talk to their doctor to learn if they have an
indicated CF gene mutation. It is not known if SYMDEKO is safe and
effective in children under 6 years of age.
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 12 years and older who have
at least one copy of the F508del mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. Patients should
talk to their doctor to learn if they have an indicated CF gene
mutation. It is not known if TRIKAFTA is safe and effective in
children under 12 years of age.
Patients should not take KALYDECO, SYMDEKO, or TRIKAFTA 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, SYMDEKO, or TRIKAFTA, patients should
tell their doctor about all of their medical conditions, including
if they: have kidney problems; have or have had liver problems;
are pregnant or plan to become pregnant because it is not known if
KALYDECO, SYMDEKO, or TRIKAFTA will harm an unborn baby; or are
breastfeeding or planning to breastfeed because it is not known if
KALYDECO, SYMDEKO, or TRIKAFTA passes into breast milk. Before
taking KALYDECO, patients should tell their doctor if they drink
grapefruit juice or eat grapefruit or Seville oranges.
KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other
medicines work, and other medicines may affect how KALYDECO,
SYMDEKO, or TRIKAFTA work. Therefore, the dose of KALYDECO,
SYMDEKO, or TRIKAFTA 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, SYMDEKO, or TRIKAFTA 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, SYMDEKO, or TRIKAFTA affects them.
Patients should avoid food or drink containing grapefruit
or Seville oranges while taking KALYDECO. Patients should
avoid food or drink containing grapefruit while taking SYMDEKO
or TRIKAFTA.
KALYDECO, SYMDEKO, and TRIKAFTA can cause serious side
effects, such as:
High liver enzymes in the blood have been reported in
patients receiving KALYDECO, SYMDEKO, or TRIKAFTA. The
patient's doctor will do blood tests to check their liver before
starting treatment with KALYDECO, SYMDEKO, or TRIKAFTA, every 3
months during the first year of treatment, and every year while on
treatment. 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) in some children
and adolescents treated with KALYDECO, SYMDEKO, or TRIKAFTA. If the
patient is a child or adolescent, their doctor should perform eye
examinations before and during treatment with KALYDECO, SYMDEKO, or
TRIKAFTA to look for cataracts.
The most common side effects of KALYDECO 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.
The most common side effects of SYMDEKO include headache,
nausea, sinus congestion, and dizziness.
The most common side effects of TRIKAFTA include
headache, diarrhea, upper respiratory tract infection (common cold)
including stuffy and runny nose, stomach (abdominal) pain, inflamed
sinuses, increase in liver enzymes, increase in a certain blood
enzyme called creatine phosphokinase, rash, flu (influenza), and
increase in blood bilirubin.
These are not all the possible side effects of KALYDECO,
SYMDEKO, or TRIKAFTA. Please click the product link to see the
full Prescribing Information for KALYDECO, SYMDEKO, or
TRIKAFTA.
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, statements made by Dr. Altshuler in
this press release and statements regarding our expectations
relating to the potential approval of TRIKAFTA, SYMDEKO, and
KALYDECO for additional CFTR mutations, the FDA's target action
date and information regarding the review process in the United
States, the data supporting product approval, and the number of
additional patients that may benefit from our medicines. 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 regulatory authorities may not approve, or
approve on a timely basis, the three sNDAs, that data from the
company's submissions 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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InvestorInfo@vrtx.com or 617-961-7163
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
International: +44 20 3204 5275
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