- First regulatory authorization of a
CRISPR-based gene-editing therapy in the world –
- CASGEVY is indicated for the treatment of
sickle cell disease in patients 12 years of age and older with
recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0
genotype, for whom hematopoietic stem cell transplantation is
appropriate and a human leukocyte antigen matched related
hematopoietic stem cell donor is not available -
- CASGEVY is indicated for the treatment of
transfusion‑dependent beta thalassemia in patients 12 years of age
and older for whom hematopoietic stem cell transplantation is
appropriate and a human leukocyte antigen matched related
hematopoietic stem cell donor is not available -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
Therapeutics (Nasdaq: CRSP) announced today that the United Kingdom
(U.K.) Medicines and Healthcare products Regulatory Agency (MHRA)
has granted conditional 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 has been authorized for the treatment of eligible
patients 12 years of age and older with SCD with recurrent
vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte
antigen (HLA) matched related hematopoietic stem cell donor is not
available. There are an estimated 2,000 patients eligible for
CASGEVY in the U.K.
“Today is a historic day in science and medicine: this
authorization of CASGEVY in Great Britain is the first regulatory
authorization of a CRISPR-based therapy in the world,” said Reshma
Kewalramani, M.D., Chief Executive Officer and President of
Vertex.
“I hope this represents the first of many applications of this
Nobel Prize winning technology to benefit eligible patients with
serious diseases,” said Samarth Kulkarni, Ph.D., Chairman and Chief
Executive Officer of CRISPR Therapeutics.”
In two global clinical trials of CASGEVY in SCD and TDT, the
trials met their respective primary outcome of becoming free from
severe VOCs or transfusion independent for at least 12 consecutive
months. Once achieved, these benefits are potentially expected to
be life-long. The safety profile of 97 SCD and TDT patients treated
to date with CASGEVY in these ongoing studies is generally
consistent with myeloablative conditioning with busulfan and
hematopoietic stem cell transplant.
“This authorization offers a new option for eligible patients
who are waiting for innovative therapies, and I look forward to
patients having access to this therapy as quickly as possible,”
said Professor Josu de la Fuente, Principal Investigator in the
CLIMB-111 and CLIMB-121 studies, Professor of Practice (Cellular
& Gene Therapy) at Imperial College London, and Consultant
Haematologist at Imperial College Healthcare NHS Trust.
In the U.K., exa-cel was granted an Innovation Passport under
the Innovative Licensing and Access Pathway (ILAP) from the MHRA,
and Vertex is already working closely with national health
authorities to secure access for eligible patients as quickly as
possible.
About Sickle Cell Disease Sickle cell disease (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” blood cells. People with SCD can
experience painful blood vessel blockages, also known as
vaso-occlusive crises (VOCs), that can lead to acute chest
syndrome, stroke, jaundice and symptoms of heart failure.
Individuals may also experience anemia, which can result in
end-organ damage and premature death. VOCs are the hallmark of SCD,
often resulting in severe and debilitating pain. Current standard
treatment options for SCD are largely symptomatic treatments and do
not adequately address the burden of disease or alleviate the need
for chronic care. Most often, treatment is focused on relieving
pain, minimizing organ damage, maintaining hydration and addressing
fevers, requiring medication and sometimes monthly blood
transfusions and frequent hospital visits. The only cure for SCD
today is a stem cell transplant from a matched donor, but this
option is only available to a small fraction of people living with
SCD. SCD requires lifelong treatment and significant use of health
care resources, and ultimately results in reduced life expectancy
and reduced lifetime earnings and productivity. In the U.K., the
mean age of death for people living with SCD is around 40.
About Beta Thalassemia Beta thalassemia 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. A lack of red blood cells, also known as anemia, is the
primary manifestation of beta thalassemia. Because of this anemia,
people living with beta thalassemia may experience fatigue and
shortness of breath, and infants may develop failure to thrive,
jaundice and feeding problems. Complications of beta thalassemia
can also include an enlarged spleen, liver and/or heart; misshapen
bones; and delayed puberty. Treatment for beta thalassemia is
personalized and depends on the severity of disease that each
person experiences. Many people have to get regular blood
transfusions to deliver healthy donated blood to their body. This
requires many hospital visits and can also lead to an unhealthy
buildup of iron. Today, stem cell transplant from a matched donor
is a curative option but is only available to a small fraction of
people living with beta thalassemia. Beta thalassemia 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
the U.K., the mean age of death for people living with TDT is
around 55.
About CASGEVY™ (exagamglogene autotemcel [exa-cel])
CASGEVY™ is a genetically modified autologous CD34+ cell enriched
population that contains human hematopoietic stem and progenitor
cells edited ex vivo by CRISPR/Cas9 at the erythroid-specific
enhancer region of the BCL11A gene.
The latest data from the ongoing pivotal trials was presented at
the European Hematology Association Congress in June 2023.
Exa-cel is also under review by the European Medicines Agency,
the Saudi Food and Drug Authority, and the U.S. Food and Drug
Administration (FDA). The FDA has granted Priority Review for SCD
and Standard Review for TDT and assigned Prescription Drug User Fee
Act (PDUFA) target action dates of December 8, 2023, and March 30,
2024, respectively.
About Conditional Marketing Authorizations Conditional
marketing authorizations (CMAs) are for medicines that fulfill 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 it is judged that such data will become
available soon. CMAs are valid for one year and renewable annually
with ongoing regulatory review of data.
About the Vertex and CRISPR Collaboration Vertex and
CRISPR Therapeutics entered into a strategic research collaboration
in 2015 focused on the use of CRISPR/Cas9 to discover and develop
potential new treatments aimed at the underlying genetic causes of
human disease. Exa-cel represents the first treatment to emerge
from the joint research program. Under an amended collaboration
agreement, Vertex now leads global development, manufacturing and
commercialization of exa-cel and splits program costs and profits
worldwide 60/40 with CRISPR Therapeutics.
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.
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 based in Boston, Massachusetts and San
Francisco, California, and business offices in London, United
Kingdom. For more information, please visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and design logo are
trademarks and registered trademarks of CRISPR Therapeutics AG. All
other trademarks and registered trademarks are the property of
their respective owners.
(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., Samarth Kulkarni, Ph.D., and Professor Josu de
la Fuente in this press release, and statements regarding our
expectations for and the anticipated benefits of CASGEVY, including
the expectation for certain life-long benefits of CASGEVY for
patients, the estimated eligible patient population in the U.K.,
Vertex’s efforts to secure access for eligible patients as quickly
as possible, and Vertex’s plans and expectations for the ongoing
clinical trials evaluating exa-cel. 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, and other
reasons, that obtaining authorization and commercializing exa-cel
in Europe, the Kingdom of Saudi Arabia, and the U.S. 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 MHRA’s 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.
(CRSP-GEN)
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 by Reshma
Kewalramani, M.D., Samarth Kulkarni, Ph.D., and Professor Josu de
la Fuente in this press release, as well as statements regarding:
(i) plans and expectations for the commercialization of, and
anticipated benefits of, CASGEVY, including the longevity of such
benefits for patients, the estimated eligible patient population in
the U.K., and the speed by which access for eligible patients may
be secured; (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) expectations for the
benefits of CRISPR Therapeutics’ collaboration with 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
authorization or renewal of conditional authorization; adequate
pricing or reimbursement may not be secured to support continued
development or commercialization of exa-cel following regulatory
authorization; 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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version on businesswire.com: https://www.businesswire.com/news/home/20231115290500/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
CRISPR Therapeutics Investors: Susan Kim
+1-617-315-4600 susan.kim@crisprtx.com
Media: Rachel Eides +1-617-315-4493
rachel.eides@crisprtx.com
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