– First-ever approval of a CRISPR-based
gene-editing therapy in the U.S. –
– Approximately 16,000 patients 12 years of age
and older with severe sickle cell disease may now be eligible for
this one-time treatment –
– Multiple authorized treatment centers
activated –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and
Drug Administration (FDA) has approved CASGEVY™ (exagamglogene
autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy,
for the treatment of sickle cell disease (SCD) in patients 12 years
and older with recurrent vaso-occlusive crises (VOCs). This
approval means that for the first time, approximately 16,000
patients with SCD may be eligible for a durable one-time therapy
that offers the potential of a functional cure for their disease by
eliminating severe VOCs and hospitalizations caused by severe
VOCs.
“CASGEVY’s approval by the FDA is momentous: it is the first
CRISPR-based gene-editing therapy to be approved in the U.S. As
importantly, CASGEVY is a first-in-class treatment that offers the
potential of a one-time transformative therapy for eligible
patients with sickle cell disease,” said Reshma Kewalramani, M.D.,
Chief Executive Officer and President of Vertex. “I want to convey
my deepest gratitude to the patients and investigators whose trust
in this program paved the way for this landmark approval.”
“When our company was founded, we had a vision to translate
CRISPR technology into multiple breakthrough therapies. So, this
U.S. approval of the first-ever medicine using CRISPR gene editing
is breathtaking, and a truly humbling moment for me personally and
for the whole organization,” said Samarth Kulkarni, Ph.D., Chairman
and Chief Executive Officer of CRISPR Therapeutics.
“It has been remarkable to be part of this groundbreaking
program,” said Stephan Grupp, M.D., Ph.D., Section Chief of the
Cellular Therapy and Transplant Section and Director of the Kelly
Center for Cancer Immunotherapy at the Children's Hospital of
Philadelphia, and Steering Committee Chair for the CLIMB-121
clinical program. “CASGEVY has the potential to be a transformative
treatment for patients, and I look forward to continuing the work
to ensure eligible patients can access this therapy across the
country.”
The administration of CASGEVY requires specialized experience in
stem cell transplantation; therefore, Vertex is engaging with
experienced hospitals to establish a network of independently
operated, authorized treatment centers (ATCs) throughout the U.S.
to offer CASGEVY to patients. The following ATCs are already
activated:
- Boston Medical Center in Boston, Mass.
- Children’s National Hospital in Washington, D.C.
- City of Hope Children’s Cancer Center in Los Angeles,
Calif.
- Medical City Children’s Hospital in Dallas, Texas
- Methodist Children’s Hospital in San Antonio, Texas
- Nationwide Children’s Hospital in Columbus, Ohio
- The Children’s Hospital at TriStar Centennial in Nashville,
Tenn.
- The Ohio State University Comprehensive Cancer Center – James
Cancer Hospital and Solove Research Institute in Columbus,
Ohio
- University of Chicago/Comer Children’s Hospital in Chicago,
Ill.
Additional ATCs will be activated in the coming weeks and a
complete list of ATCs, including updates following approval, can be
accessed at CASGEVY.com.
About CASGEVY™ (exagamglogene autotemcel [exa-cel])
CASGEVY is a genome-edited cellular therapy consisting of
autologous CD34+ hematopoietic stem cells (HSCs) edited by
CRISPR/Cas9 technology at the erythroid-specific enhancer region of
the BCL11A gene. CASGEVY is intended for one time administration
via a hematopoietic stem cell transplant procedure where the
patient’s own CD34+ cells are modified to reduce BCL11A expression
in erythroid lineage cells, leading to increased fetal hemoglobin
(HbF) production. 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 vaso-occlusive crises for
patients with SCD.
CASGEVY was granted a conditional marketing authorization in
Great Britain by the U.K. Medicines and Healthcare products
Regulatory Agency and by the National Health Regulatory Authority
in Bahrain for patients 12 years of age and older with SCD
characterized by recurrent vaso-occlusive crises or
transfusion-dependent beta thalassemia (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 European
Medicines Agency and the Saudi Food and Drug Agency for both SCD
and TDT.
The use of CASGEVY for the treatment of TDT in the U.S. remains
investigational. Vertex has submitted a BLA to the U.S. FDA for the
potential use of CASGEVY for patients 12 years and older with TDT
and has been assigned a Prescription Drug User Fee Act (PDUFA)
target action date of March 30, 2024.
About the Vertex/CRISPR Collaboration Financial
Reporting Vertex leads global development, manufacturing
and commercialization of CASGEVY with support from CRISPR
Therapeutics. In conjunction with the FDA approval of CASGEVY,
Vertex will make a $200 million milestone payment to CRISPR, which
will be capitalized and amortized to cost of sales. Additionally,
Vertex will record 100 percent of CASGEVY revenues; costs of sales;
and selling, general and administrative expenses and will record
CRISPR’s 40 percent share in the net profits or losses from CASGEVY
within its cost of sales. Lastly, Vertex will record 60 percent of
research and development expenses, net of CRISPR’s 40 percent
share.
About Sickle Cell Disease Sickle cell disease (SCD)
is a debilitating, progressive and life-shortening disease. SCD
patients report health-related quality of life scores well below
the general population, and the lifetime health care costs in the
U.S. of managing SCD for patients with recurrent VOCs is estimated
between $4 and $6 million. SCD is an inherited blood disorder that
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 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 a lifetime of
treatment and results in a reduced life expectancy. In the U.S.,
the median age of death for patients living with SCD is
approximately 45 years. A cure for SCD today is a stem cell
transplant from a matched donor, but this option is only available
to a small fraction of patients living with SCD because of the lack
of available donors.
Helping Patients on the CASGEVY Journey Vertex
Connects™ is a program for eligible patients in the U.S. who have
been prescribed CASGEVY. Through Vertex Connects, Care Managers are
available to provide educational resources, communications and
support to navigate the treatment journey, and additional
assistance is available for eligible patients.
For more information on ATCs, the CASGEVY treatment journey, and
Vertex Connects, call 1-833-VERTEX-5 (1-833-837-8395) — and learn
more about our patient support program at VertexConnects.com.
U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY
(exagamglogene autotemcel)
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 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)
(CRSP-GEN)
About CRISPR Therapeutics CRISPR Therapeutics is a
leading gene editing company focused on developing transformative
gene-based medicines for serious diseases using its proprietary
CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing
technology that allows for precise, directed changes to genomic
DNA. CRISPR Therapeutics has established a portfolio of therapeutic
programs across a broad range of disease areas including
hemoglobinopathies, oncology, regenerative medicine and rare
diseases. To accelerate and expand its efforts, CRISPR Therapeutics
has established strategic collaborations with leading companies
including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR
Therapeutics AG is headquartered in Zug, Switzerland, with its
wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and
R&D operations in Boston, Massachusetts and San Francisco,
California, and business offices in London, United Kingdom. For
more information, please visit www.crisprtx.com.
CRISPR THERAPEUTICS® word mark and design are trademarks and
registered trademarks of CRISPR Therapeutics AG. All other
trademarks and registered trademarks are the property of their
respective owners.
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., Samarth Kulkarni, Ph.D., and Stephan Grupp,
M.D., Ph.D., and statements regarding the anticipated patient
population eligible for CASGEVY in the U.S., expectations for the
potential benefits of CASGEVY, expectations that additional ATCs
will be activated in the coming weeks, Vertex’s plan to make
milestone payments to CRISPR, and Vertex’s anticipated accounting
treatment for milestone payments, anticipated revenue, the cost of
sales, expenses, and net profits or losses related to CASGEVY.
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 approval for and
commercializing CASGEVY in Europe and the Kingdom of Saudi Arabia
may not occur 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 or the scientific data
presented. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
CRISPR Therapeutics Forward-Looking Statement
This press release may contain a number of “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended, including statements made by Reshma
Kewalramani, M.D., Samarth Kulkarni, Ph.D., and Stephan Grupp,
M.D., Ph.D., in this press release, as well as statements regarding
CRISPR Therapeutics’: (i) plans and expectations for the
commercialization of, and anticipated benefits of, CASGEVY,
including the anticipated patient population eligible for CASGEVY
in the United States and patient access to CASGEVY; (ii)
expectations regarding the ongoing exa-cel clinical trials,
including potential implications of clinical data for patients;
(iii) timelines for and expectations regarding additional
regulatory agency decisions; (iv) receipt of the milestone payment
from Vertex; and (v) expectations regarding the therapeutic value,
development, and commercial potential of CRISPR/Cas9 gene editing
technologies and therapies. Without limiting the foregoing, the
words “believes,” “anticipates,” “plans,” “expects” and similar
expressions are intended to identify forward-looking statements.
You are cautioned that forward-looking statements are inherently
uncertain. Although CRISPR Therapeutics believes that such
statements are based on reasonable assumptions within the bounds of
its knowledge of its business and operations, existing and
prospective investors are cautioned that forward-looking statements
are inherently uncertain, are neither promises nor guarantees and
not to place undue reliance on such statements, which speak only as
of the date they are made. Actual performance and results may
differ materially from those projected or suggested in the
forward-looking statements due to various risks and uncertainties.
These risks and uncertainties include, among others, that: the
clinical data from ongoing clinical trials of exa-cel will not
continue or be repeated in ongoing or planned clinical trials or
may not support regulatory approval or renewal of conditional
authorization; regulatory approval in other jurisdictions may not
occur on anticipated timelines or at all; adequate pricing or
reimbursement may not be secured to support continued development
or commercialization of exa-cel following regulatory approval;
future competitive or other market factors may adversely affect the
commercial potential for CASGEVY; CRISPR Therapeutics may not
realize the potential benefits of its collaboration with Vertex;
there are uncertainties regarding the intellectual property
protection for CRISPR Therapeutics’ technology and intellectual
property belonging to third parties; and those risks and
uncertainties described under the heading “Risk Factors” in CRISPR
Therapeutics’ most recent annual report on Form 10-K, quarterly
report on Form 10-Q, and in any other subsequent filings made by
CRISPR Therapeutics with the U.S. Securities and Exchange
Commission, which are available on the SEC's website at
www.sec.gov. CRISPR Therapeutics disclaims any obligation or
undertaking to update or revise any forward-looking statements
contained in this press release, other than to the extent required
by law.
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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
CRISPR Therapeutics Investors: Susan Kim, +1 617-307-7503
susan.kim@crisprtx.com
Media: Rachel Eides, +1-617-315-4493
rachel.eides@crisprtx.com
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