- Approximately 300 more people with cystic
fibrosis in the U.S. are now eligible for a medicine that treats
the underlying cause of their disease for the first time -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the U.S. Food and Drug Administration (FDA) has approved
the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) for the treatment of people with cystic fibrosis (CF)
ages 2 and older who have at least one F508del mutation in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene or
a mutation that is responsive to TRIKAFTA based on clinical and/or
in vitro data. In addition, safety information on liver injury and
liver failure has been updated from warnings and precautions to a
boxed warning. With this approval, 94 additional non-F508del CFTR
mutations have been added to the TRIKAFTA label, and approximately
300 additional people with CF in the U.S. are now eligible for a
medicine to treat the underlying cause of their disease for the
first time.
“Since its first approval in 2019, TRIKAFTA has had a
transformative impact on tens of thousands of people living with
cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice
President, Global Medicines Development and Medical Affairs, and
Chief Medical Officer, Vertex. “With this approval, even more
patients may be able to benefit from a medicine that treats the
underlying cause of their disease, and we look forward to
continuing the work to extend the approvals and availability of our
medicines to patients around the world.”
For more information on TRIKAFTA, patient assistance programs or
to find additional eligibility details, visit TRIKAFTA.com,
VertexGPS.com or vertextreatments.com.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 92,000 people globally. CF is a progressive,
multi-organ disease that affects the lungs, liver, pancreas, GI
tract, sinuses, sweat glands 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, and these mutations
can be identified by a genetic test. While there are many different
types of CFTR mutations that can cause the disease, the vast
majority of people with CF have at least one F508del mutation. CFTR
mutations lead to CF by causing CFTR protein to be defective or by
leading to a shortage or absence of CFTR protein 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, chronic lung infections and
progressive lung damage that eventually leads to death for many
patients. The median age of death is in the 30s, but with
treatment, projected survival is improving.
Today Vertex CF medicines are treating over 68,000 people with
CF across more than 60 countries on six continents. This represents
2/3 of the diagnosed people with CF eligible for CFTR modulator
therapy.
About TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and
ivacaftor)
In people with certain types of mutations in the CFTR gene, the
CFTR protein is not processed or folded normally within the cell,
and this can prevent the CFTR protein from reaching the cell
surface and functioning properly. TRIKAFTA
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral
medicine designed to increase the quantity and function of the CFTR
protein at the cell surface. Elexacaftor and tezacaftor work
together to increase the amount of mature protein at the cell
surface. Ivacaftor, which is known as a CFTR potentiator, is
designed to facilitate the ability of CFTR proteins to transport
salt and water across the cell membrane. The combined actions of
elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus
from the airways.
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a
prescription medicine used for the treatment of cystic fibrosis
(CF) in patients aged 2 years and older who have at least one copy
of the F508del mutation or a mutation in the CFTR gene that is
responsive based on clinical and/or in vitro data. 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 2 years of age.
The following 94 mutations have been added to the TRIKAFTA®
label for the first time:
1507_1515del9, 2183A→G, A1067P, A107G, A309D, A62P, C491R,
D1445N, D565G, D993Y, E116Q, E292K, F1107L, F200I, F587I, G1047R,
G1123R, G1247R, G27E, G424S, G480S, G551A, G970S, H620P, H620Q,
H939R;H949L, I105N, I125T, I148N, I331N, I506L, I556V, K162E,
K464E, L1011S, L137P, L333F, L333H, L441P, L619S, M1137V, M150K,
N1088D, N1303I, N186K, N187K, N418S, P140S, P499A, P750L, Q1313K,
Q372H, Q493R, Q552P, R1048G, R117C;G576A;R668C, R297Q, R31C, R516S,
R555G, R709Q, R75L, S1045Y, S108F, S1118F, S1235R, S549I, T1086I,
T1246I, T1299I, T351I, V392G, V603F, Y301C, 2789+5G→A, 3272-26A→G,
3849+10kbC→T, N1303K, 711+3A→G, E831X, 5T;TG12, 5T;TG13, 296+28A→G,
621+3A→G, 1898+3A→G, 2789+ 2insA, 3850-3T→G, 3600G→A, 3849+4A→G,
3849+40A→G, 4005+2T→C, 1341G→A, 3041-15T→G, 2752-26A→G
TRIKAFTA U.S. INDICATIONS
TRIKAFTA is indicated for the treatment of cystic fibrosis (CF)
in patients aged 2 years and older who have at least one F508del
mutation in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene or a mutation in the CFTR gene that is responsive based
on clinical and/or in vitro data.
If the patient’s genotype is unknown, an FDA-cleared CF mutation
test should be used to confirm the presence of at least one
indicated mutation.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: DRUG-INDUCED LIVER INJURY AND LIVER
FAILURE
TRIKAFTA can cause serious and potentially fatal drug-induced
liver injury. Cases of liver failure leading to transplantation and
death have been reported in patients with and without a history of
liver disease taking TRIKAFTA, in both clinical trials and the
post-marketing setting. Liver injury has been reported within the
first month of therapy and up to 15 months following initiation of
TRIKAFTA.
Assess liver function tests (ALT, AST, alkaline phosphatase,
and bilirubin) in all patients prior to initiating TRIKAFTA, then
every month during the first 6 months of treatment, every 3 months
for the next 12 months, and at least annually thereafter. Consider
more frequent monitoring for patients with a history of liver
disease or liver function test (LFT) elevations at
baseline.
Interrupt TRIKAFTA for significant elevations in LFTs or in
the event of signs or symptoms of liver injury. Consider referral
to a hepatologist. Follow patients closely with clinical and
laboratory monitoring until abnormalities resolve. If resolved,
resume treatment only if the benefit is expected to outweigh the
risk. Closer monitoring is advised after resuming TRIKAFTA.
TRIKAFTA should not be used in patients with severe hepatic
impairment (Child-Pugh Class C). TRIKAFTA is not recommended in
patients with moderate hepatic impairment (Child-Pugh Class B). If
used, use with caution at a reduced dosage and monitor patients
closely.
WARNINGS AND PRECAUTIONS
Drug-Induced Liver Injury and Liver Failure
- TRIKAFTA can cause serious and potentially fatal drug-induced
liver injury. Liver failure leading to transplantation and death
have been reported in patients with and without a history of liver
disease taking TRIKAFTA. Liver injury has been reported within the
first month of therapy and up to 15 months following initiation of
TRIKAFTA
- Assess LFTs (ALT, AST, alkaline phosphatase, and bilirubin) in
all patients prior to initiating TRIKAFTA, then every month during
the first 6 months of treatment, every 3 months for the next 12
months, at least annually thereafter
- Interrupt TRIKAFTA in the event of signs or symptoms of liver
injury, which may include:
- Significant elevations in LFTs (e.g. ALT or AST >5x the
upper limit of normal (ULN) or ALT or AST >3x ULN with bilirubin
>2x ULN)
- Clinical symptoms suggestive of liver injury (e.g., jaundice,
right upper quadrant pain, nausea, vomiting, altered mental status,
ascites)
- Consider referral to a hepatologist and follow patients closely
with clinical and laboratory monitoring until the abnormalities
resolve. If resolved, and if benefit is expected to outweigh risk,
resume TRIKAFTA with close monitoring
- TRIKAFTA should not be used in patients with severe hepatic
impairment . TRIKAFTA is not recommended in patients with moderate
hepatic impairment and should only be considered when there is a
clear medical need, and benefit outweighs risk. If used, use with
caution at a reduced dosage and monitor patients closely
Hypersensitivity Reactions, Including Anaphylaxis
- Hypersensitivity reactions, including cases of angioedema and
anaphylaxis, have been reported in the post-marketing setting. If
signs or symptoms of serious hypersensitivity reactions develop
during treatment, discontinue TRIKAFTA and institute appropriate
therapy. Consider benefits and risks for the individual patient to
determine whether to resume treatment with TRIKAFTA
Concomitant Use With CYP3A Inducers
- Exposure to ivacaftor is significantly decreased and exposure
to elexacaftor and tezacaftor are expected to decrease by
concomitant use of strong CYP3A inducers, which may reduce the
therapeutic effectiveness of TRIKAFTA. Concomitant use with strong
CYP3A inducers is not recommended
Concomitant Use With CYP3A Inhibitors
- Exposure to elexacaftor, tezacaftor, and ivacaftor are
increased when used concomitantly with strong or moderate CYP3A
inhibitors. The dose of TRIKAFTA should be reduced when used
concomitantly with moderate or strong CYP3A inhibitors
Cataracts
- Non-congenital lens opacities have been reported in pediatric
patients treated with ivacaftor-containing regimens. Baseline and
follow-up ophthalmological examinations are recommended in
pediatric patients initiating treatment with TRIKAFTA
ADVERSE REACTIONS
Serious Adverse Reactions
- Serious adverse reactions that occurred more frequently in
patients treated with TRIKAFTA compared to placebo were rash (1% vs
<1%) and influenza (1% vs 0%)
Most Common Adverse Reactions
- The most common adverse reactions occurring in ≥5% of patients
treated with TRIKAFTA and higher than placebo by ≥1% were headache,
upper respiratory tract infection, abdominal pain, diarrhea, rash,
alanine aminotransferase increased, nasal congestion, blood
creatine phosphokinase increased, aspartate aminotransferase
increased, rhinorrhea, rhinitis, influenza, sinusitis, and blood
bilirubin increased and constipation
USE IN SPECIFIC POPULATIONS
Pediatric Use
- The safety and effectiveness of TRIKAFTA in patients with CF
younger than 2 years of age have not been established
Please click here to see the full U.S. Prescribing
Information, including Boxed WARNING for 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 approved medicines that
treat the underlying causes of multiple chronic, life-shortening
genetic diseases — cystic fibrosis, sickle cell disease and
transfusion-dependent beta thalassemia — and continues to advance
clinical and research programs in these diseases. Vertex also has a
robust clinical pipeline of investigational therapies across a
range of modalities in other serious diseases where it has deep
insight into causal human biology, including acute and neuropathic
pain, APOL1-mediated kidney disease, IgA nephropathy, primary
membranous nephropathy, autosomal dominant polycystic kidney
disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international headquarters in London. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia, Latin America and the
Middle East. Vertex is consistently recognized as one of the
industry's top places to work, including 15 consecutive years on
Science magazine's Top Employers list and one of Fortune’s 100 Best
Companies to Work For. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, Facebook, Instagram, YouTube and X.
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 Carmen Bozic,
M.D., in this press release, statements regarding the eligible
patient population for TRIKAFTA, expectations for access to
TRIKAFTA for eligible patients, and statements regarding the
potential benefits of TRIKAFTA. 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 risks listed
under the heading “Risk Factors” in Vertex's annual report and in
subsequent filings filed with the Securities and Exchange
Commission and available through the company's website at
www.vrtx.com and www.sec.gov. You should not place undue reliance
on these statements. 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 Incorporated Investors:
investorsinfo@vrtx.com Susie Lisa, CFA: +1 617-341-6108 or Manisha
Pai: +1 617-961-1899
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 Heather
Nichols: +1 617-839-3607 or International: +44 20 3204 5275
Vertex Pharmaceuticals (NASDAQ:VRTX)
過去 株価チャート
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Vertex Pharmaceuticals (NASDAQ:VRTX)
過去 株価チャート
から 1 2024 まで 1 2025
