- Reimbursement agreement also includes certain
future indication extensions across all Vertex CF medicines -
- Approximately 1,400 patients will now have
access to a CFTR modulator for the first time -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced it has reached a new portfolio agreement with the Italian
Medicines Agency, AIFA, for the reimbursement of all of Vertex’s
approved medicines for the treatment of cystic fibrosis (CF),
including KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
combination regimen with ivacaftor.
Italian patients ages 12 years and older with one F508del
mutation and one minimal function mutation (F/MF) or two F508del
mutations (F/F) in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene will now have access to KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with
ivacaftor. Additionally, under the terms of the agreement, eligible
patients ages two years and older with CF who have two copies of
the F508del mutation in the CFTR gene (F/F) will now have access to
ORKAMBI® (lumacaftor/ivacaftor). Eligible patients ages 12 years
and older who either have two copies of the F508del mutation (F/F),
or one copy of the F508del mutation and another responsive residual
function mutation in the CFTR gene (F/RF), will have broad access
to SYMKEVI® (tezacaftor/ivacaftor) in combination with ivacaftor.
The agreement also expands access to KALYDECO® (ivacaftor) for
eligible patients ages one year and older. In addition, the
agreement covers any new approved indication extensions for
Vertex’s CF medicines submitted and approved for reimbursement
during the term of the contract.
Ludovic Fenaux, Senior Vice President, Vertex International,
commented, “This agreement is an important milestone for cystic
fibrosis patients in Italy. Our medicines have fundamentally
changed the way CF is treated, and we are delighted with this broad
portfolio agreement which includes access for younger patients to
ORKAMBI and KALYDECO and access to SYMKEVI and KAFTRIO for patients
12 years and older. I would like to thank AIFA and all parties
involved for their collaboration, commitment and engagement in
quickly reaching this agreement.”
KALYDECO® was first reimbursed in Italy in 2015, followed by
ORKAMBI® in 2017 for patients 12 years and older. Vertex’s CF
medicines are reimbursed in over 25 countries around the world
including Australia, France, Germany, the Republic of Ireland, the
Netherlands, Spain, Sweden, the U.K. and the U.S.
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 ORKAMBI® (lumacaftor/ivacaftor) and the F508del
Mutation
In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little-to-no CFTR protein at the cell surface.
Patients with two copies of the F508del mutation are easily
identified by a simple genetic test.
Lumacaftor/ivacaftor is a combination of lumacaftor, which is
designed to increase the amount of mature protein at the cell
surface by targeting the processing and trafficking defect of the
F508del-CFTR protein, and ivacaftor, which is designed to enhance
the function of the CFTR protein once it reaches the cell
surface.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About KALYDECO® (ivacaftor)
Ivacaftor is the first medicine to treat the underlying cause of
CF in people with specific mutations in the CFTR gene. Known as a
CFTR potentiator, ivacaftor is an oral medicine designed to keep
CFTR proteins at the cell surface open longer to improve the
transport of salt and water across the cell membrane, which helps
hydrate and clear mucus from the airways.
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 Ludovic Fenaux,
Senior Vice President, Vertex International, in this press release
and statements regarding our beliefs about the eligible patient
population that will have access to our medicines, including
patients that will now have access to a CFTR modulator for the
first time, reimbursement coverage for future approved indication
extensions of our medicines, 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 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 an extended indication for our medicines, 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.
(VRTX-GEN)
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