- If approved by the European Commission,
patients 12 years of age and older with severe sickle cell disease
or transfusion-dependent beta thalassemia, for whom hematopoietic
stem cell transplantation is appropriate and a human leukocyte
antigen matched related donor is not available, would be eligible
for treatment -
- Approval decision from the European
Commission is expected in Q1 2024 -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced
today that the European Medicines Agency’s (EMA’s) Committee for
Medicinal Products for Human Use (CHMP) has adopted a positive
opinion for the conditional approval of CASGEVY™ (exagamglogene
autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the
treatment of severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT).
If approved, exa-cel would be the only genetic therapy for
patients in the European Union who are 12 years of age and older
with either severe SCD with recurrent vaso-occlusive crises (VOCs)
or TDT, for whom hematopoietic stem cell (HSC) transplantation is
appropriate and a human leukocyte antigen matched related HSC donor
is not available. An approval decision by the European Commission
is expected in February 2024.
“This positive opinion is yet another important regulatory
milestone underscoring the potentially transformative benefit of
CASGEVY for eligible patients with sickle cell and
transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D.,
Executive Vice President and Chief Regulatory and Quality Officer
at Vertex.
“There is an urgent need for new potentially curative treatments
in beta thalassemia and sickle cell disease, as people with these
diseases still have a shorter life expectancy than the general
population and an impaired quality of life,” 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.
“As an investigator, I have witnessed first-hand the transformative
impact exa-cel can have on patients’ lives and I eagerly await the
approval in the European Union.”
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 alleviate
transfusion requirements for patients with TDT.
The use of CASGEVY in the European Union remains
investigational.
CASGEVY is approved in the U.S. to treat people aged 12 years
and older with SCD who have recurrent VOCs. CASGEVY was granted a
conditional marketing authorization in Great Britain by the U.K.
Medicines and Healthcare products Regulatory Agency for patients 12
years of age and older with SCD characterized by recurrent VOCs or
TDT, for whom hematopoietic stem cell transplantation is
appropriate and a human leukocyte antigen matched related
hematopoietic stem cell donor is not available. CASGEVY is
currently under review by the Saudi Food and Drug Authority for
both SCD and TDT and the U.S. Food and Drug Administration for
TDT.
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.
U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY
(exagamglogene autotemcel)
WHAT IS CASGEVY?
CASGEVY is a one-time therapy used to treat people aged 12 years
and older with sickle cell disease (SCD) who have frequent
vaso-occlusive crises or VOCs.
CASGEVY is made specifically for each patient, using the
patient’s own edited blood stem cells, and increases the production
of a special type of hemoglobin called hemoglobin F (fetal
hemoglobin or HbF). Having more HbF increases overall hemoglobin
levels and has been shown to improve the production and function of
red blood cells. This can eliminate VOCs in people with SCD.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
CASGEVY? After treatment with CASGEVY, you will have fewer
blood cells for a while until CASGEVY takes hold (engrafts) into
your bone marrow. This includes low levels of platelets (cells that
usually help the blood to clot) and white blood cells (cells that
usually fight infections). Your doctor will monitor this and give
you treatment as required. The doctor will tell you when blood cell
levels return to safe levels.
- Tell your healthcare provider right away if you
experience any of the following, which could be signs of low levels
of platelet cells:
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums;
blood in your urine, stool, or vomit; or coughing up blood
- Tell your healthcare provider right away if you
experience any of the following, which could be signs of low levels
of white blood cells:
You may experience side effects associated with other medicines
administered as part of the treatment regimen with CASGEVY. Talk to
your physician regarding those possible side effects. Your
healthcare provider may give you other medicines to treat your side
effects.
How will I receive CASGEVY? Your healthcare provider will
give you other medicines, including a conditioning medicine, as
part of your treatment with CASGEVY. It's important to talk to your
healthcare provider about the risks and benefits of all medicines
involved in your treatment.
After receiving the conditioning medicine, it may not be
possible for you to become pregnant or father a child. You should
discuss options for fertility preservation with your healthcare
provider before treatment.
STEP 1: Before CASGEVY treatment, a doctor will give you
a mobilization medicine. This medicine moves blood stem cells from
your bone marrow into the blood stream. The blood stem cells are
then collected in a machine that separates the different blood
cells (this is called apheresis). This entire process may happen
more than once. Each time, it can take up to one week.
During this step, rescue cells are also collected and stored at
the hospital. These are your existing blood stem cells and are kept
untreated just in case there is a problem in the treatment process.
If CASGEVY cannot be given after the conditioning medicine, or if
the modified blood stem cells do not take hold (engraft) in the
body, these rescue cells will be given back to you. If you are
given rescue cells, you will not have any treatment benefit from
CASGEVY.
STEP 2: After they are collected, your blood stem cells
will be sent to the manufacturing site where they are used to make
CASGEVY. It may take up to 6 months from the time your cells are
collected to manufacture and test CASGEVY before it is sent back to
your healthcare provider.
STEP 3: Shortly before your stem cell transplant, your
healthcare provider will give you a conditioning medicine for a few
days in hospital. This will prepare you for treatment by clearing
cells from the bone marrow, so they can be replaced with the
modified cells in CASGEVY. After you are given this medicine, your
blood cell levels will fall to very low levels. You will stay in
the hospital for this step and remain in the hospital until after
the infusion with CASGEVY.
STEP 4: One or more vials of CASGEVY will be given into a
vein (intravenous infusion) over a short period of time.
After the CASGEVY infusion, you will stay in hospital so that
your healthcare provider can closely monitor your recovery. This
can take 4-6 weeks, but times can vary. Your healthcare provider
will decide when you can go home.
What should I avoid after receiving CASGEVY?
- Do not donate blood, organs, tissues, or cells at any time in
the future
What are the possible or reasonably likely side effects of
CASGEVY?
The most common side effects of CASGEVY include:
- Low levels of platelet cells, which may reduce the ability of
blood to clot and may cause bleeding
- Low levels of white blood cells, which may make you more
susceptible to infection
Your healthcare provider will test your blood to check for low
levels of blood cells (including platelets and white blood cells).
Tell your healthcare provider right away if you get any of the
following symptoms:
- fever
- chills
- infections
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums;
blood in your urine, stool, or vomit; or coughing up blood
These are not all the possible side effects of CASGEVY. Call
your doctor for medical advice about side effects. You may report
side effects to FDA at 1-800-FDA-1088.
General information about the safe and effective use of
CASGEVY
Talk to your healthcare provider about any health concerns.
Please see full Prescribing Information including Patient
Information for CASGEVY.
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 cause 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 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 and Latin America.
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, Facebook, Instagram, 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, statements made by Nia
Tatsis, Ph.D., and Franco Locatelli, M.D., Ph.D., in this press
release and statements regarding our expectations for regulatory
approval for CASGEVY™, including anticipated timing of approval,
the anticipated population eligible for treatment, our expectation
that, if approved, CASGEVY will be the only genetic therapy
available for eligible patients in the European Union, and our
beliefs regarding the benefits of our genetic therapy. 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 CASGEVY on a timely basis or at all,
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231214351567/en/
Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com or Manisha Pai, +1 617-961-1899
Media:
mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.:
617-341-6992 or Heather Nichols: +1 617-839-3607
Vertex Pharmaceuticals (NASDAQ:VRTX)
Gráfico Histórico do Ativo
De Mai 2024 até Jun 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Gráfico Histórico do Ativo
De Jun 2023 até Jun 2024