Designation based on Phase II SPRINT trial
in pediatric patients with NF1 plexiform neurofibromas
Selumetinib is a MEK 1/2 Inhibitor being
co-developed by AstraZeneca and Merck
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known
as MSD outside the US and Canada) today announced that the US Food
and Drug Administration (FDA) has granted Breakthrough Therapy
Designation (BTD) for the investigational MEK 1/2 inhibitor and
potential new medicine selumetinib.
This designation is for the treatment of pediatric patients aged
three years and older with neurofibromatosis type 1 (NF1)
symptomatic and/or progressive, inoperable plexiform neurofibromas
(PN), a rare, incurable genetic condition.
José Baselga, Executive Vice President, Research and
Development, Oncology, said: “Selumetinib shows promise in the
treatment of NF1-related plexiform neurofibromas, a rare and
debilitating disease with no approved medications to date. The
Breakthrough Therapy Designation acknowledges the significant unmet
need of these patients and the potential benefit of selumetinib in
this setting.”
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, at Merck Research Laboratories,
said: “This new designation validates our ongoing development of
selumetinib. As a result of this, selumetinib has the potential to
receive expedited regulatory review and we hope to bring this
medicine to patients as soon as possible.”
The BTD is based on Phase II data from the SPRINT trial, testing
selumetinib as an oral monotherapy in pediatric patients, aged
three years or older with inoperable NF1-related PN. The results of
the trial were presented by the National Cancer Institute (NCI) at
the 2018 American Society of Clinical Oncology Annual Meeting.
This is the ninth BTD that AstraZeneca has received from the FDA
since 2014. A BTD is designed to expedite the development and
regulatory review of medicines that are intended to treat a serious
condition and that have shown encouraging early clinical results,
which may demonstrate substantial improvement on a
clinically-significant endpoint over available medicines.
Selumetinib was granted Orphan Drug Designation for the
treatment of NF1 by the US FDA in February 2018 and the European
Medicines Agency in August 2018.
About selumetinib
Selumetinib is a MEK 1/2 inhibitor and potential new medicine
licensed by AstraZeneca from Array BioPharma Inc. in 2003.
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, entered a
co-development and co-commercialization agreement for selumetinib
in 2017.
The NF1 gene provides instructions for making a protein called
neurofibromin, which negatively regulates the RAS/MAPK pathway,
helping to control cell growth, differentiation and survival.
Mutations in the NF1 gene may result in dysregulations in
RAS/RAF/MEK/ERK signaling, which can cause cells to grow, divide
and copy themselves in an uncontrolled manner, and may result in
tumor growth. Selumetinib inhibits the MEK enzyme in this pathway,
potentially leading to inhibition of tumor growth.
Selumetinib is being assessed as a monotherapy and in
combination with other treatments in ongoing clinical trials.
About SPRINT
The SPRINT trial is a US Cancer Therapy Evaluation Program
(CTEP) NCI-sponsored Phase I/II trial. The Phase I trial was
designed to identify the optimal Phase II dosing regimen, and the
results were published in the New England
Journal of Medicine.
About NF1
Neurofibromatosis type 1 (NF1) is an incurable genetic condition
that affects one in 3,000 to 4,000 individuals in the U.S. It is
caused by a spontaneous or inherited mutation in the NF1 gene and
is associated with many symptoms, including soft lumps on and under
the skin (cutaneous neurofibromas), skin pigmentation (so-called
‘cafe au lait’ spots) and, in 20-50% of patients, tumors develop on
the nerve sheaths (plexiform neurofibromas).These plexiform
neurofibromas can cause clinical complications such as pain, motor
dysfunction, airway dysfunction, bowel/bladder dysfunction and
disfigurement as well as having the potential to transform into
malignant peripheral nerve sheath tumors (MPNST).
People with NF1 may experience a number of complications such as
learning difficulties, visual impairment, twisting and curvature of
the spine, high blood pressure, and epilepsy. NF1 also increases a
person’s risk of developing other cancers, including malignant
brain tumors, MPNST and leukemia. Symptoms begin during early
childhood, with varying degrees of severity, and can reduce life
expectancy by up to 15 years.
About the AstraZeneca and the Merck Oncology
Collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth,
NJ, US, known as MSD outside the United States and Canada,
announced a global strategic oncology collaboration to co-develop
and co-commercialize LYNPARZA (olaparib), the world’s first PARP
inhibitor and potential new medicine selumetinib, a MEK inhibitor,
for multiple cancer types. Working together, the companies will
develop LYNPARZA and selumetinib in combination with other
potential new medicines and as monotherapies. Independently, the
companies will develop LYNPARZA and selumetinib in combination with
their respective PD-L1 and PD-1 medicines.
About AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in Oncology and offers a
quickly-growing portfolio of new medicines that has the potential
to transform patients’ lives and the Company’s future. With at
least six new medicines to be launched between 2014 and 2020, and a
broad pipeline of small molecules and biologics in development, we
are committed to advance New Oncology as one of AstraZeneca’s five
Growth Platforms focused on lung, ovarian, breast and blood
cancers. In addition to our core capabilities, we actively pursue
innovative partnerships and investments that accelerate the
delivery of our strategy, as illustrated by our investment in
Acerta Pharma in hematology.
By harnessing the power of four scientific platforms –
Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response
and Antibody Drug Conjugates – and by championing the development
of personalized combinations, AstraZeneca has the vision to
redefine cancer treatment and one day eliminate cancer as a cause
of death.
About AstraZeneca
AstraZeneca is a global, science-led biopharmaceutical company
that focuses on the discovery, development and commercialization of
prescription medicines, primarily for the treatment of diseases in
three therapy areas - Oncology, Cardiovascular, Renal &
Metabolism and Respiratory. AstraZeneca operates in over 100
countries and its innovative medicines are used by millions of
patients worldwide. For more information, please
visit www.astrazeneca-us.com and follow us on Twitter
@AstraZenecaUS.
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