Vertex Announces European Commission Approval for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor ...
April 28 2021 - 7:30AM
Business Wire
- New indication includes people ages 12 years
and older who have one copy of the F508del mutation regardless of
the other mutation type -
- People with gating (F/G) or residual function
(F/RF) mutations now eligible for the triple combination therapy
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted approval of the
label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in
a combination regimen with ivacaftor for the treatment of cystic
fibrosis (CF) in all patients ages 12 years and older who have at
least one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. With this extension of the
indication, CF patients 12 years and older who are heterozygous for
the F508del-CFTR mutation and a residual function (F/RF) or gating
mutation (F/G) are eligible for the triple combination therapy for
the first time, and the majority of people ages 12 years and older
with CF in Europe are now eligible for KAFTRIO.
“This indication extension is important as CF patients in Europe
with gating and residual function mutations will have access to
KAFTRIO for the first time,” said Reshma Kewalramani, M.D., Chief
Executive Officer and President at Vertex. “We look forward to
working with health authorities and governments to ensure that all
eligible patients who can benefit from this medicine have access as
soon as possible.”
In both the United States and Australia, where the triple
combination therapy is known as TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor), it is already
licensed for the treatment of people with CF ages 12 years and
older who have at least one copy of the F508del mutation in the
CFTR gene, regardless of the other mutation type.
“In clinical trials ivacaftor/tezacaftor/elexacaftor plus
ivacaftor showed positive significant results in people with cystic
fibrosis ages 12 years and older who have at least one copy of the
most common gene mutation (F508del) with the latest results showing
clinical benefits in individuals who have an additional ‘gating’
(F/G) or ‘residual function’ (F/RF) gene mutation. It is welcome
news for the CF community that the European regulatory authority
has extended the licensed indications for this therapy based on the
latest trial results which means that additional patients will gain
access to this medication,” said Dr Peter Barry, Honorary Senior
Lecturer at The University of Manchester.
As a result of long-term reimbursement agreements in the
Republic of Ireland, Northern Ireland, Denmark and Luxembourg, and
provisions for access in health care systems such as Germany,
eligible patients in these countries will have access to the
expanded indication for the triple combination regimen shortly
following regulatory approval by the European Commission.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 80,000 people globally.1 CF is a progressive,
multi-system disease that affects the lungs, liver,
gastrointestinal (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.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
Combination With Ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination
regimen with ivacaftor 150 mg was developed for the treatment of
cystic fibrosis (CF) in patients ages 12 years and older who have
at least one copy of the F508del mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene.
Ivacaftor/tezacaftor/elexacaftor is designed to increase the
quantity and function of the F508del-CFTR protein at the cell
surface. The latest approved EU licensed indication for
ivacaftor/tezacaftor/elexacaftor was supported by positive results
of three global Phase 3 studies in people ages 12 years and older
with CF: a 24-week Phase 3 study (Study 445-102) in 403 people with
one F508del mutation and one minimal function mutation (F/MF), a
four-week Phase 3 study (Study 445-103) in 107 people with two
F508del mutations (F/F), and a Phase 3 study (Study 445-104) in 258
people heterozygous for the F508del-CFTR mutation and a CFTR gating
mutation (F/G) or a residual function mutation (F/RF).2
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
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 cell and genetic 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. 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 11 consecutive years on Science magazine's Top
Employers list and a best place to work for LGBTQ equality by the
Human Rights Campaign.
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. Reshma
Kewalramani and Dr. Peter Barry in this press release, statements
regarding the eligible patient population in Europe and our
expectations regarding the timing of access to KAFTRIO® in
combination with ivacaftor for eligible patients ages 12 years and
older in Europe. 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, risks related to commercializing KAFTRIO® in
combination with ivacaftor in Europe, and other risks listed under
the heading “Risk Factors” in Vertex's annual report filed with the
Securities and Exchange Commission and available through the
company's website at www.vrtx.com and on the SEC’s website at
www.sec.gov. You should not place undue reliance on these
statements or the scientific data presented. Vertex disclaims any
obligation to update the information contained in this press
release as new information becomes available.
(VRTX-GEN)
1 Vertex. Data on File. Global CF EPI. 2021. 2 EMA. Kaftrio
Summary of product characteristics. Vertex Pharmaceuticals
(Ireland) Limited. Available at:
https://www.ema.europa.eu/en/documents/product-information/kaftrio-epar-product-information_en.pdf
Last accessed April 2021.
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