- Over 8,000 patients 12 years of age and older
with severe sickle cell disease or transfusion-dependent beta
thalassemia may be eligible for treatment -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced
today that the European Commission has granted conditional
marketing authorization to CASGEVY™ (exagamglogene autotemcel
[exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved
for the treatment of patients who are 12 years of age and older
with severe sickle cell disease (SCD) characterized by recurrent
vaso-occlusive crises (VOCs) or transfusion-dependent beta
thalassemia (TDT), for whom hematopoietic stem cell (HSC)
transplantation is appropriate and a human leukocyte antigen
matched related HSC donor is not available.
CASGEVY is the only genetic therapy approved for SCD and TDT
patients in the European Union (EU) and with this approval, there
are now more than 8,000 patients potentially eligible for
treatment.
“With this approval, CASGEVY is now approved for sickle cell
disease and transfusion-dependent beta thalassemia in multiple
geographies making tens of thousands of patients eligible for this
potentially transformative therapy,” said Reshma Kewalramani, M.D.,
Chief Executive Officer and President of Vertex. “Now our goal
shifts to translating these approvals into real-world patient
benefit and ensuring access and reimbursement across the
globe.”
“Sickle cell disease and transfusion-dependent beta thalassemia
are debilitating, life-shortening diseases associated with
significant burden on patients, families and health care systems,”
said Franco Locatelli, M.D., Ph.D., Principal investigator in the
CLIMB-111 and CLIMB-121 studies, Professor of Pediatrics at the
Catholic University of the Sacred Heart, Rome, and Director of the
Department of Pediatric Hematology and Oncology at the Bambino Gesù
Children’s Hospital. “CASGEVY offers the potential of a functional
cure, and it will be important to offer this therapeutic option to
eligible patients as soon as possible.”
Vertex is already working closely with national health
authorities to secure access for eligible patients as quickly as
possible. Through this work, Vertex has secured early access for
eligible TDT patients in France ahead of the national reimbursement
process. Vertex continues to engage with hospitals experienced in
stem cell transplantation to establish a network of independently
operated authorized treatment centers (ATCs) for the administration
of CASGEVY. There are currently three activated ATCs in the EU and
Vertex plans to activate a total of approximately 25 centers across
Europe.
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. In Europe, the mean age of death for
patients living with SCD is around 40 years. Stem cell transplant
from a matched donor is a curative option but is only available to
a small fraction of people living with SCD because of the lack of
available donors.
About Transfusion-Dependent Beta Thalassemia (TDT)
TDT is a serious, life-threatening 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. In
Europe, the mean age of death for patients living with TDT is 50-55
years. Stem cell transplant from a matched donor is a curative
option but is only available to a small fraction of people living
with TDT because of the lack of available donors.
About CASGEVY™ (exagamglogene autotemcel [exa-cel])
CASGEVY™ 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. CASGEVY has been shown to
reduce or eliminate VOCs for patients with SCD and transfusion
requirements for patients with TDT.
CASGEVY is approved for certain indications in multiple
jurisdictions for eligible patients.
About Conditional Marketing Authorizations (CMAs)
CMAs are for medicines that fulfil a significant unmet medical
need such as being for serious and life-threatening diseases, where
no satisfactory treatment methods are available or where the
medicine offers a major therapeutic advantage. A CMA is granted
where comprehensive clinical data is not yet complete, but the
benefit of the medicine to address a significant unmet need
outweighs the need for data that will become available in the
future. CMAs are valid for one year and renewable annually with
ongoing regulatory review of data.
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 APOL1-mediated kidney
disease, acute and neuropathic pain, 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.com 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, as
amended, including, without limitation, the statements by Reshma
Kewalramani, M.D., and Franco Locatelli, M.D., Ph.D., in this press
release, and statements regarding our expectations for and the
anticipated benefits of CASGEVY, the estimated eligible patient
population, our efforts to secure access for eligible patients as
quickly as possible, our continued engagement with hospitals to
establish a network of ATCs and our plans to activate a total of
approximately 25 centers across Europe. While we believe 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, and other
reasons, that commercializing CASGEVY in multiple geographies may
not occur on the anticipated timeline, or at all, that adequate
pricing and reimbursement for CASGEVY may not be achieved on the
anticipated timeline, or at all, that the conditional marketing
authorization may not be renewed annually, or at all, 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 at www.sec.gov and available
through the company's website at www.vrtx.com. 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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5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607
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