- CASGEVY™ is first medicine ever to be
evaluated through the SFDA’s Breakthrough Medicines Program -
- First treatment center in Saudi Arabia has
been activated -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced
today that the Saudi Food and Drug Authority (SFDA) granted
Marketing Authorization for CASGEVY™ (exagamglogene autotemcel
[exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of
sickle cell disease (SCD) and transfusion-dependent beta
thalassemia (TDT). CASGEVY is approved for the treatment of people
12 years of age and older with SCD or TDT. The Kingdom of Saudi
Arabia has among the highest prevalence rates of SCD and TDT in the
world, with thousands of patients living with these genetic blood
disorders.
“This approval adds to the list of firsts for CASGEVY. It
represents the first medicine ever to receive SFDA Breakthrough
Designation and be approved through this pathway, and Vertex’s
first ever regulatory approval in the Kingdom of Saudi Arabia,”
said Reshma Kewalramani, M.D., Chief Executive Officer and
President of Vertex. “Most importantly, with this approval, people
living with sickle cell disease or transfusion-dependent beta
thalassemia have the possibility of a one-time transformative
therapy for their disease.”
The Ministry of National Guard Health Affairs (MNGHA) is the
first Authorized Treatment Center (ATC) in Saudi Arabia. Vertex is
working to qualify additional hospitals as ATCs to bring CASGEVY to
patients, including the King Faisal Specialist Hospital (KFSH). In
order to enable rapid access to CASGEVY, Vertex is working to
secure listing on hospital formularies to support reimbursement as
soon as possible.
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 a 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 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. 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.
CASGEVY is approved for certain indications in multiple
jurisdictions for eligible patients.
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 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, 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, the statements by Reshma
Kewalramani, M.D., and statements regarding the expectations for
the potential benefits of CASGEVY, our plans to work to enable
rapid access to CASGEVY, including our plans to secure listing on
hospital formularies to support reimbursement as soon as possible,
and our work to qualify additional hospitals as ATCs to bring
CASGEVY to patients, including the KFSH. 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, that obtaining additional approvals and/or 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,
and other risks listed under the heading “Risk Factors” in Vertex's
most recent annual report and subsequent filings 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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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
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