Vertex Announces National Reimbursement Agreement in France for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) & SYMKEVI® (tez...
June 28 2021 - 9:26AM
Business Wire
- With this
reimbursement agreement more than 1,500 patients now have access to
a CFTR modulator therapy for the first time -
Vertex Pharmaceuticals
Incorporated (Nasdaq:
VRTX) today announced a national reimbursement agreement with the
French Health Authorities for the cystic fibrosis (CF) medicines
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination
regimen with ivacaftor and SYMKEVI® (tezacaftor/ivacaftor) in
combination with ivacaftor. Both medicines will be available for
all eligible patients once the agreement has been published in the
French Official Journal.
“Today’s announcement represents a major milestone for CF
patients in France. Through this national reimbursement agreement,
eligible patients 12 years and older now have access to KAFTRIO and
SYMKEVI. For those living with CF, we are delighted to have reached
this agreement so quickly and that the French Health Authorities
have recognized the value of both medicines,” said Ludovic Fenaux,
Senior Vice President, Vertex International.
The reimbursement agreement enables broad access to KAFTRIO® for
people with CF ages 12 years and older with one F508del mutation
and one minimal function mutation. The triple combination therapy
will also be reimbursed for patients who are homozygous for the
F508del mutation in the CFTR gene, representing a new therapeutic
option for treating physicians. In November 2020, the Transparency
Commission (TC) of the French National Authority for Health (HAS)
granted KAFTRIO® an ASMR 2 rating, which indicates “a significant
improvement in medical service rendered.” Of the 250 first-time
listing medicines issued in 2019 by the TC, only two received such
a rating.
Under the terms of the new reimbursement agreement, SYMKEVI®
will be reimbursed for people with CF ages 12 years and older with
one F508del mutation and one of the mutations resulting in residual
activity (F/RF) of the CFTR protein as listed in the Summary of
Product Characteristics (SmPC). It will also be funded for patients
who are homozygous for the F508del mutation in the CFTR gene.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 80,000 people globally. 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.
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).
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About SYMKEVI® (tezacaftor/ivacaftor) in Combination With
Ivacaftor
Some mutations result in CFTR protein that is not processed or
folded normally within the cell, and that generally does not reach
the cell surface. Tezacaftor is designed to address the trafficking
and processing defect of the CFTR protein to enable it to reach the
cell surface and ivacaftor is designed to enhance the function of
the CFTR protein once it reaches the cell surface.
For complete product information including dosing guidance,
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 Ludovic Fenaux,
Senior Vice President, Vertex International in this press release
and statements regarding the reimbursement of, availability of and
access to KAFTRIO® and SYMKEVI® for certain patients, the estimated
number of patients eligible for a CFTR modulator therapy and our
beliefs regarding the benefits of 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 risks and uncertainties that could cause
actual events or results to differ materially from those expressed
or implied 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 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.
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