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 February 2018
Commission
File Number: 001-11960
AstraZeneca PLC
1
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AstraZeneca PLC
INDEX
TO EXHIBITS
Selumetinib
in NF1 gets FDA orphan drug status
15 February 2018 07:00 GMT
SELUMETINIB GRANTED ORPHAN DRUG DESIGNATION BY THE US FDA FOR
NEUROFIBROMATOSIS TYPE 1
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 Orphan Drug Designation (ODD) for
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
births,[i]
with highly-variable symptoms, including cutaneous (skin),
neurological (nervous system) and orthopaedic (skeletal)
manifestations. NF1 can cause secondary complications including
learning difficulties, visual impairment, pain, disfigurement,
twisting and curvature of the spine, high blood pressure and
epilepsy. Plexiform neurofibromas (PNs) are a neurological
manifestation of NF1 and arise from nerve fascicles that tend to
grow along the length of the nerve. PNs occur in approximately
20-50% of NF1 patients causing pain, motor dysfunction and
disfigurement.[ii]
Sean Bohen, Executive Vice President, Global Medicines Development
and Chief Medical Officer, at AstraZeneca, said: "Neurofibromatosis
type 1 is a devastating condition that can lead to life-threatening
complications. There is no known cure for neurofibromatosis and
there are limited treatment options to manage
symptoms."
Roy Baynes, Senior Vice President and Head of Global
Clinical Development, Chief Medical Officer, at MSD Research
Laboratories, said: "We're looking forward to working with our
colleagues at AstraZeneca to develop selumetinib and understand how
it may benefit patients with NF1."
The potential benefit of selumetinib in NF1 is being explored in
the US National Cancer Institute-sponsored Phase I/II SPRINT trial in paediatric patients
with symptomatic NF1-related PNs. Phase II trial results are
expected later in 2018.
The
FDA's ODD programme provides orphan status to medicines that are
defined as those intended for the safe and effective treatment,
diagnosis or prevention of rare diseases or disorders that affect
fewer than 200,000 people in the US.
In
addition to NF1, selumetinib is being investigated in the Phase III
ASTRA trial of patients who are diagnosed with differentiated
thyroid cancer (DTC) following surgery and treatment with
radioactive iodine. Selumetinib was granted ODD by the US FDA for
the adjuvant treatment of stage III/IV DTC in 2016. It is also
being explored as a monotherapy and in combination with other
treatments in Phase I trials.
About neurofibromatosis type 1 (NF1)
NF1 is
caused by a spontaneous or inherited mutation in the NF1 gene. 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, tumours on the
nerve sheaths (plexiform neurofibromas). These plexiform
neurofibromas can cause morbidities such as pain, motor dysfunction
and disfigurement. Patients 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. People with NF1 also have an increased 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.[iii]
About selumetinib
Selumetinib
is an investigational MEK 1/2 inhibitor licensed by AstraZeneca
from Array BioPharma Inc. in 2003.
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.
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
(olaparib), the world's first PARP inhibitor, and potential new
medicine selumetinib, a MEK inhibitor, for multiple cancer types.
The collaboration is based on increasing evidence that PARP and MEK
inhibitors can be combined with PD-L1/PD-1 inhibitors for a range
of tumour types. Working together, the companies will develop
Lynparza and selumetinib in
combination with other potential new medicines and as a
monotherapy. 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 & Metabolic
Diseases and Respiratory. The Company also is selectively active in
the areas of autoimmunity, neuroscience and infection. 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
[i] 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.
[ii] Dombi E,
et
al. Activity of Selumetinib in
Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
N Engl J
Med. 2016;
375:2550-2560.
[iii] Evans DGR,
et
al. Reduced Life Expectancy
Seen in Hereditary Diseases Which Predispose to Early-Onset
Tumors. Appl Clin Genet.
2013; 6:53-61.
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:
15 February
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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