a4970r
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 June
2020
Commission
File Number: 001-11960
AstraZeneca PLC
1
Francis Crick Avenue
Cambridge
Biomedical Campus
Cambridge
CB2 0AA
United
Kingdom
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Selumetinib
granted orphan drug designation in Japan for neurofibromatosis type
1
30 June 2020 07:00 BST
Selumetinib granted orphan drug designation
in Japan for neurofibromatosis type 1
Designation follows recent US approval
with additional regulatory submissions underway
Phase II SPRINT trial showed selumetinib reduced
tumour
volume in paediatric patients with NF1 plexiform
neurofibromas
AstraZeneca today announced that selumetinib has been granted
orphan drug designation (ODD) in Japan for the treatment of
neurofibromatosis type 1 (NF1), a rare and debilitating genetic
disease.1
Selumetinib is co-developed and co-commercialised with MSD Inc.,
Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside
the US and Canada).
Some 30-50% of patients with NF1 experience plexiform neurofibromas
(PN) - tumours growing along their nerve
sheaths.2 These
PN can cause clinical issues such as disfigurement, motor
dysfunction, pain, airway dysfunction, visual impairment and
bowel/bladder dysfunction.3
The Japanese Ministry of Health, Labour and Welfare grants ODD to
medicines intended for the treatment of diseases that affect fewer
than 50,000 patients in Japan and for which there is a high unmet
medical need.
José Baselga, Executive Vice President, Oncology R&D,
said: "Neurofibromatosis type 1 can have a devastating impact on
children and new medicines are urgently needed to help treat the
resulting plexiform neurofibromas and associated clinical issues.
Current options in most countries are limited and this designation
is a significant step forward in bringing the first medicine for
NF1 to paediatric patients in Japan."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories,
said: "Plexiform neurofibromas are one of the key
manifestations of NF1 and can lead to pain and disfigurement. In
the SPRINT trial, selumetinib was shown to reduce the size of these
tumours in children. We are hopeful that we will be able to bring
this treatment to this underserved paediatric patient community in
Japan."
The National Cancer Institute (NCI) Cancer Therapy Evaluation
Program (CTEP)-sponsored Phase I/II SPRINT Stratum 1 trial showed
an overall response rate (ORR) of 66% (33 of 50 patients, confirmed
partial response) in paediatric patients with NF1 PN when treated
with selumetinib as a twice-daily oral monotherapy. ORR is defined
as the percentage of patients with confirmed complete or partial
response of at least 20% reduction in tumour volume.
AstraZeneca and MSD are jointly developing and commercialising
selumetinib which was approved in the US in
April 2020 under the
medicine name Koselugo for the treatment of paediatric patients two
years and older with NF1 and symptomatic, inoperable PN. A
marketing authorisation application in NF1 PN was accepted for
review by the European Medicines Agency earlier in the year and
further global regulatory submissions are
underway.
NF1
NF1 is a debilitating genetic disease that affects one in every
3,000 to 4,000 individuals.1 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) and skin pigmentation
(so-called 'café au lait' spots)1 and,
in 30-50% of patients, tumours develop on the nerve sheaths
(plexiform neurofibromas).2 These
plexiform neurofibromas can cause clinical issues such as
disfigurement, motor dysfunction, pain, airway dysfunction, visual
impairment, and bladder/bowel dysfunction.3 PN
begin during early childhood, with varying degrees of severity, and
can reduce life expectancy by 8 to 15 years.1,4,5
SPRINT
The SPRINT Stratum 1 Phase I/II trial was designed to evaluate the
objective response rate and impact on patient-reported and
functional outcomes in paediatric patients with NF1-related
inoperable PNs treated with selumetinib
monotherapy.6 Results
were published in The New
England Journal of Medicine.7 This
trial sponsored by NCI CTEP was conducted under a Cooperative
Research and Development Agreement between NCI and AstraZeneca with
additional support from the Neurofibromatosis Therapeutic
Acceleration Program (NTAP).
Selumetinib
Selumetinib (available in the US under the medicine
name Koselugo) is an inhibitor of mitogen-activated protein
kinase kinases 1 and 2 (MEK1/2).6 MEK1/2
proteins are upstream regulators of the extracellular
signal-related kinase (ERK) pathway. Both MEK and ERK are critical
components of the RAS-regulated RAF-MEK-ERK pathway, which is often
activated in different types of cancers.
Selumetinib was granted US FDA Breakthrough Therapy
Designation in April 2019,
Rare Pediatric Disease Designation in December 2019, Orphan Drug
Designation in February 2018, EU orphan
designation in August 2018
and Swissmedic orphan drug status in December 2018 for the
treatment of paediatric patients with NF1 PN.
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 Koselugo, a MEK inhibitor, for multiple
cancer types. Working together, the companies will
develop Lynparza and Koselugo in combination with other potential
new medicines and as monotherapies. Independently, the companies
will develop Lynparza and Koselugo in combination with their respective
PD-L1 and PD-1 medicines.
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 six new
medicines launched between 2014 and 2020, and a broad pipeline of
small molecules and biologics in development, the Company is
committed to advance oncology as a key growth driver for
AstraZeneca focused on lung, ovarian, breast and blood cancers. In
addition to AstraZeneca's main capabilities, the Company is
actively pursuing innovative partnerships and investments that
accelerate the delivery of our strategy, as illustrated by the
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.
AstraZeneca
AstraZeneca (LSE/STO/NYSE: AZN) 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
& Immunology. Based in Cambridge, UK, AstraZeneca operates in
over 100 countries and its innovative medicines are used by
millions of patients worldwide. Please
visit astrazeneca.com and
follow the Company on Twitter @AstraZeneca.
Contacts
For details on how to contact the Investor Relations Team, please
click here.
For Media contacts, click here.
References
1. National Institute
of Neurological Disorders and Stroke. Neurofibromatosis Fact Sheet.
"What is NF1?". Available at: https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet
#3162_2 Accessed February
2020.
2. Hirbe AC, Gutmann DH.
Neurofibromatosis type 1: a multidisciplinary approach to
care. The Lancet
Neurology. 2014;13:834-43.
DOI: 10.1016/S1474-4422(14)70063-8.
3. Dombi E, Baldwin A, Marcus
LJ, et
al. Activity of selumetinib in
neurofibromatosis type 1-related plexiform
neurofibromas. N Engl J
Med. 2016;375:2550-2560. DOI:
10.1056/NEJMoa1605943.
4. Rasmussen SA, Yang Q, Friedman JM.
Mortality in neurofibromatosis 1: an analysis using U.S. death
certificates. Am J Hum
Genet.
2001;68:1110-1118.
5. Evans DGR, O'Hara C, Wilding
A, et
al. Mortality in
neurofibromatosis 1: in North West England: an assessment of
actuarial survival in a region of the UK since
1989. Eur J Hum
Genet. 2011;19:1187-1191.
DOI: 10.1038/ejhg.2011.113.
6. Koselugo (selumetinib) [prescribing information].
Wilmington, DE: AstraZeneca Pharmaceuticals LP;
2020.
7. Gross A, Wolters P, Dombi
E, et
al. Selumetinib in Children
with Inoperable Plexiform Neurofibromas. N Engl J
Med. 2020;382: DOI:
10.1056/NEJMoa11912735.
Adrian Kemp
Company Secretary
AstraZeneca PLC
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:
30 June
2020
|
By: /s/
Adrian Kemp
|
|
Name:
Adrian Kemp
|
|
Title:
Company Secretary
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