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AstraZeneca: 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 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

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

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

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
Company Secretary
AstraZeneca PLC


1 NHS Choices. Neurofibromatosis Type 1. Available at
https://www.nhs.uk/conditions/neurofibromatosis-type-1/. Accessed May

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.

3. Evans DGR, et al. Reduced Life Expectancy Seen in Hereditary
Diseases Which Predispose to Early-Onset Tumors. Appl Clin Genet.
2013; 6:53-61.


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