-Exa-cel is the first CRISPR-based gene-edited
therapy to be submitted for Health Canada review-
TORONTO, April 1,
2024 /CNW/ - Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced its New Drug
Submission (NDS) for exagamglogene autotemcel (exa-cel) has been
accepted for Priority Review by Health Canada for the treatment of
patients aged 12 years and older with sickle cell disease (SCD)
with recurrent vaso-occlusive crises (VOCs) and for the treatment
of patients aged 12 years and older with transfusion-dependent beta
thalassemia (TDT).
"We are pleased that exa-cel has been accepted for Priority
Review by Health Canada and look forward to bringing this therapy
to eligible patients," said Michael
Siauw, General Manager at Vertex Pharmaceuticals
(Canada) Incorporated.
The NDS will be part of an aligned review with Health Technology
Assessment (HTA) organizations, the Canadian Agency for Drugs and
Technologies in Health (CADTH) and the Institut national
d'excellence en santé et en services sociaux (INESSS) in
Quebec.
With Priority Review, the conventional review timeline of 300
days is reduced to 180 days.
The NDS is supported by results from the ongoing Phase 3
studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term
follow-up study, CLIMB-131. Data from the Phase 3 studies were most
recently presented at the American Society of
Hematology (ASH) Annual Meeting and Exposition in December of
2023.
About exagamglogene autotemcel (exa-cel)
Exa-cel, formerly known as CTX001, is a non-viral, ex
vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients
with SCD or TDT, in which a patient's own hematopoietic stem and
progenitor cells are edited at the erythroid specific enhancer
region of the BCL11A gene through a precise double-strand
break. This edit results in the production of high levels of fetal
hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form
of the oxygen-carrying hemoglobin that is naturally present during
fetal development, which then switches to the adult form of
hemoglobin after birth. Exa-cel has been shown to reduce or
eliminate VOCs for patients with SCD and transfusion requirements
for patients with TDT. Earlier results from these ongoing trials
were published in The New England Journal of
Medicine in January of 2021 and presented at the ASH
Annual Meeting and Exposition in December 2023.
Exa-cel remains investigational in Canada and the safety and efficacy has not
been established by Health Canada. Exa-cel is approved as CASGEVY®
for certain indications in the United
States, European Union, Great
Britain, Kingdom of Saudi
Arabia, and Bahrain.
About CLIMB-111 and CLIMB-121
The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and
CLIMB-121, are designed to assess the safety and efficacy of a
single dose of exa-cel in patients ages 12 to 35 years with TDT or
with SCD, characterized by recurrent VOCs, respectively. The trials
are now closed for enrollment. Patients will be followed for
approximately two years after exa-cel infusion. Each patient will
be asked to participate in CLIMB-131, a long-term follow-up
trial.
About CLIMB-131
The ongoing long-term, open-label trial, CLIMB-131, is designed
to evaluate the safety and efficacy of exa-cel in patients who
previously received exa-cel.
About Sickle Cell Disease (SCD)
SCD is a debilitating, progressive, life shortening genetic
disease. SCD patients report health-related quality of life scores
well below the general population and significant health care
resource utilization. SCD affects the red blood cells, which are
essential for carrying oxygen to all organs and tissues of the
body. SCD causes severe pain, organ damage and shortened life span
due to misshapen or "sickled" red blood cells. The clinical
hallmark of SCD is vaso-occlusive crises (VOCs), which are caused
by blockages of blood vessels by sickled red blood cells and result
in severe and debilitating pain that can happen anywhere in the
body at any time. SCD requires lifelong treatment and significant
use of health care resources, and ultimately results in reduced
life expectancy, decreased quality of life and reduced lifetime
earnings and productivity. Stem cell transplant from a matched
donor is a curative option but is only available to a small
fraction of people living with SCD.
About Transfusion-Dependent Beta Thalassemia (TDT)
TDT is a serious, life shortening genetic disease. TDT patients
report health-related quality of life scores below the general
population and significant health care resource utilization. TDT
requires frequent blood transfusions and iron chelation therapy
throughout a person's life. Due to anemia, patients living with TDT
may experience fatigue and shortness of breath, and infants may
develop failure to thrive, jaundice and feeding problems.
Complications of TDT can also include an enlarged spleen, liver
and/or heart, misshapen bones and delayed puberty. TDT requires
lifelong treatment and significant use of health care resources,
and ultimately results in reduced life expectancy, decreased
quality of life and reduced lifetime earnings and productivity.
Stem cell transplant from a matched donor is a curative option but
is only available to a small fraction of people living with
TDT.
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, autosomal dominant polycystic
kidney disease, type 1 diabetes, myotonic dystrophy type 1 and
alpha-1 antitrypsin deficiency.
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 14
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.ca or follow us on LinkedIn, YouTube and
Twitter/X.
(VRTX-GEN)
Vertex Special Note Regarding Forward-Looking
Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
without limitation, statements made by Michael Siauw in this press release, and
statements regarding Vertex's beliefs that the NDS for exa-cel will
be part of an aligned review with HTA, CADTH and INESSS, and
Vertex's expectations that the conventional review timeline will be
reduced with Priority Review. 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 regulatory authorities may not
approve, or approve on a timely basis, our regulatory submissions
for exa-cel, that data from a limited number of patients may not be
indicative of final clinical trial results, and that data from the
company's development programs may not support registration or
further development of its compounds due to safety and/or efficacy,
or other reasons, and other risks listed under the heading "Risk
Factors" in Vertex's most recent annual report and subsequent
quarterly reports filed with the Securities and Exchange
Commission (SEC) 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. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
SOURCE Vertex Pharmaceuticals (Canada) Inc.