a4959j
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 April 2020
Commission
File Number: 001-11960
AstraZeneca PLC
1
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Koselugo (selumetinib)
approved in US for paediatric patients with
neurofibromatosis type 1 plexiform
neurofibromas
13 April 2020 07:00 BST
Koselugo (selumetinib) approved in US
for paediatric
patients with neurofibromatosis type 1 plexiform
neurofibromas
First medicine approved to treat this rare and debilitating genetic
condition
AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck
& Co., Inc. inside the US and Canada) today announced that the
US Food and Drug Administration (FDA) has approved the kinase
inhibitor Koselugo (selumetinib) for the treatment of
paediatric patients two years of age and older with
neurofibromatosis type 1 (NF1) who have symptomatic, inoperable
plexiform neurofibromas (PN).1
The approval by the FDA was based on positive results from the
National Cancer Institute (NCI) Cancer Therapy Evaluation Program
(CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the
NCI's Center for Cancer Research, Pediatric Oncology Branch. This
is the first regulatory approval anywhere in the world of a
medicine for the treatment of NF1 PN.1
NF1 is a rare and debilitating genetic
condition.2 Some
30-50% of patients with NF1 experience PN - tumours growing inside
their nerve sheaths. These PN can cause clinical issues such as
pain, motor dysfunction, airway dysfunction, bowel/bladder
dysfunction and disfigurement.2,3
Results showed an overall response rate (ORR) of 66% (33 of 50
patients, confirmed partial response) in paediatric patients with
NF1 PN when treated with Koselugo 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.
Dave Fredrickson, Executive Vice President, Oncology Business Unit,
said: "For the first time, patients and families impacted by this
incurable genetic condition have an approved medicine to treat the
resulting plexiform neurofibromas. I would like to thank our
research partners, the NCI, the Neurofibromatosis Therapeutic
Acceleration Program (NTAP), the Children's Tumor Foundation (CTF),
the NF1 patient community and, most importantly, the children,
parents and doctors who participated in the SPRINT clinical trial
programme."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories,
said: "Previously there were no medicines approved for this
disease. This approval has the potential to change
how symptomatic, inoperable NF1 plexiform
neurofibromas are treated and provides new hope for these
patients."
Brigitte C. Widemann, M.D., Principal Investigator of the SPRINT
trial and Chief, National Cancer Institute Pediatric Oncology
Branch, said: "Koselugo has made a difference for many children in
this trial. This is an important treatment advance for patients and
their families."
AstraZeneca and MSD are jointly developing and
commercialising Koselugo globally and submitted a marketing
authorisation application in NF1 PN to the European Medicines
Agency in the first quarter of 2020. Further global regulatory
submissions are being evaluated.
Priority Review Voucher
AstraZeneca has received a Priority Review Voucher
(PRV) under the Rare Pediatric Disease
Designation Program intended to encourage development of new
medicines for rare paediatric diseases. A PRV entitles the holder
to FDA Priority Review of a single New Drug Application or
Biologics License Application, which reduces the target review time
and has led to an expedited approval.
Financial considerations
In accordance with the existing collaboration agreement between
Merck and AstraZeneca, following approval and upcoming launch,
AstraZeneca will book all monotherapy Product Sales
of Koselugo; half of gross profits will be due to Merck and
will be recorded under Cost of Sales. Any potential future
sales-related milestone payments will be recorded under
Collaboration Revenue. AstraZeneca will
supply Koselugo.
NF1
NF1 is a debilitating genetic condition that affects one in every
3,000 to 4,000 individuals.4 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)2 and,
in 30-50% of patients, tumours develop on the nerve sheaths
(plexiform neurofibromas). These plexiform neurofibromas can cause
clinical issues such as disfigurement, motor dysfunction,
pain, airway dysfunction, visual impairment, and bladder/bowel
dysfunction.
PN begin during early childhood, with varying degrees of severity,
and can reduce life expectancy by up to 15
years.2,4
SPRINT
The SPRINT 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 Koselugo monotherapy.1 Results
were published in The New
England Journal of Medicine.5 This
trial sponsored by NCI CTEP was conducted under a Cooperative
Research and Development Agreement between NCI and AstraZeneca with
additional support from NTAP.
Koselugo
Koselugo (selumetinib) is
inhibitor of mitogen-activated protein kinase kinases 1 and 2
(MEK1/2). 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.
Koselugo 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 and Metabolism, and Respiratory.
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. Koselugo (selumetinib) [prescribing information].
Wilmington, DE: AstraZeneca Pharmaceuticals LP;
2020.
2. National Institute of Neurological Disorders and Stroke.
Neurofibromatosis Fact Sheet. "What is NF1?". Available
at: www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet #3162_2
Accessed February 2020.
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. 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:
14 April 2020
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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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