Blueprint
FORM 6-K
SECURITIES
AND EXCHANGE COMMISSION
Washington,
D.C. 20549
Report
of Foreign Issuer
Pursuant
to Rule 13a-16 or 15d-16 of
the
Securities Exchange Act of 1934
For the
month of (August 2018)
Commission
File Number: 001-11960
AstraZeneca PLC
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AstraZeneca PLC
INDEX
TO EXHIBITS
EMA grants OD for selumetinib in NF1
03 August 2018 07:00BST
Selumetinib granted orphan designation
in Europe for neurofibromatosis type 1
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known as
MSD outside the US and Canada) today announced that the European
Medicines Agency (EMA) has granted orphan designation to
selumetinib, a MEK 1/2 inhibitor, for the treatment of
neurofibromatosis type 1 (NF1).
NF1 is an incurable genetic condition that affects one in 3,000
newborns worldwide.1,2
The severity of signs and symptoms
associated with NF1 can be highly variable, are often
mild-to-moderate and may include skin, nerve and skeletal
manifestations. Plexiform neurofibromas (PNs) are benign tumours on
nerve sheaths that develop in 20-50% of patients, and as they
continue to increase in number and size, cause moderate-to-severe
morbidities such as pain, motor dysfunction and
disfigurement.
Sean Bohen, Executive Vice President, Global Medicines Development
and Chief Medical Officer at AstraZeneca, said: "There is no cure
for NF1, a life-long and devastating condition, and current
treatment choices for these patients are very limited. The granting
of an orphan designation is a positive step forward for children
with NF1 and their families."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories said:
"NF1 is a relatively rare disease, but can lead to life-threatening
complications in those affected by it. This underscores the
importance of this collaborative effort between MSD and our partner
AstraZeneca to help patients impacted by this debilitating genetic
condition."
The potential benefit of selumetinib in NF1 is being explored in
the Phase I/II SPRINT trial in paediatric patients with inoperable
NF1-related PNs. Select findings were presented recently at the
2018 American Society of Clinical Oncology Annual Meeting in
Chicago by the principal investigators at the National Cancer
Institute. Full results are expected later in 2018.
Orphan designation is a status assigned to a medicine intended for
use in rare diseases. To be granted orphan status by the EMA, a
medicine must be intended for the treatment, prevention or
diagnosis of a disease that is seriously debilitating/life
threatening and has a prevalence of up to five in 10,000 in the
European Union. Additionally, the intended medicine must aim to
provide significant benefit to those affected by the condition.
Orphan designation is conferred following a positive opinion by the
EMA's Committee for Orphan Medicinal Products. Selumetinib was
granted Orphan Drug Designation (ODD) by the US Food and Drug
Administration (FDA) for the treatment of NF1 in February
2018.
About selumetinib
Selumetinib
is an MEK 1/2 inhibitor and potential new medicine licensed by
AstraZeneca from Array BioPharma Inc. in 2003. AstraZeneca and
Merck & Co., Inc., Kenilworth, NJ, US entered a co-development
and co-commercialisation 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 signalling, which can cause cells to grow, divide
and copy themselves in an uncontrolled manner, and may result in
tumour growth. Selumetinib inhibits the MEK enzyme in this pathway,
potentially leading to inhibition of tumour growth. It is also
being explored as a monotherapy and in combination with other
treatments in other ongoing trials.
About neurofibromatosis type 1 (NF1)
NF1 is
caused by a spontaneous or inherited mutation in the NF1 gene and
affects approximately one in 3,000 births. The disease is
associated with many symptoms, including soft lumps on and under
the skin (subcutaneous neurofibromas), skin pigmentation (cafe au
lait spots) and, in 20-50% of patients, benign tumours on the nerve
sheaths (plexiform neurofibromas). These plexiform neurofibromas
can cause morbidities such as pain, motor dysfunction and
disfigurement.
People
with NF1 may experience a number of other 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 and peripheral nerve sheath tumours, and leukaemia. Symptoms
begin during early childhood, with varying degrees of severity, and
can reduce life expectancy by up to 15 years.3
About the AstraZeneca and MSD Strategic 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-commercialise Lynparza,
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 haematology.
By
harnessing the power of four scientific platforms -
Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response
and Antibody Drug Conjugates - and by championing the development
of personalised 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 commercialisation 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.com
and follow us on Twitter
@AstraZeneca.
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Adrian Kemp
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AstraZeneca PLC
References
1 NHS Choices. Neurofibromatosis Type 1. Available at
https://www.nhs.uk/conditions/neurofibromatosis-type-1/.
Accessed May 2018.
2
Ghalayani P, et al.
Neurofibromatosis Type I (von Recklinghausen's Disease): A Family
Case Report and Literature Review. Dent Res J. 2012;9(4):
483-488.
SIGNATURES
Pursuant
to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
Date:
03 August
2018
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By: /s/
Adrian Kemp
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Name:
Adrian Kemp
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Title:
Company Secretary
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