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 2021
Commission
File Number: 001-11960
AstraZeneca PLC
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Koselugo approved in the EU for children with NF1
22 June
2021 07:00 BST
Koselugo approved in the EU
for children with neurofibromatosis type 1 and plexiform
neurofibromas
First medicine approved in the EU to treat this rare and
debilitating genetic condition
SPRINT Phase II trial showed Koselugo reduced tumour volume,
reducing pain and improving quality of life
AstraZeneca and
MSD's Koselugo
(selumetinib) has been granted conditional approval in the European
Union (EU) for the treatment of symptomatic, inoperable plexiform
neurofibromas (PN) in paediatric patients with neurofibromatosis
type 1 (NF1) aged three years and above.
NF1 is a debilitating genetic condition affecting one in 3,000
individuals worldwide.1,2
In 30-50% of people with NF1, tumours
develop on the nerve sheaths (plexiform neurofibromas) and can
cause clinical issues such as disfigurement, motor dysfunction,
pain, airway dysfunction, visual impairment and bladder or bowel
dysfunction.3-7
The
approval by the European
Commission was based on positive results from the SPRINT
Stratum 1 Phase II trial sponsored by the National Institute of
Health's National Cancer Institute (NCI) Cancer Therapy Evaluation
Program (CTEP). This trial showed Koselugo reduced the size of inoperable
tumours in children, reducing pain and improving quality of
life.7,8
This is the first approval of a medicine for NF1 PN in the EU and
follows the positive recommendation
by the Committee for Medicinal Products for Human Use of the
European Medicines Agency in April 2021. Safety and efficacy data from the SPRINT Phase II
trial with longer follow up will be provided as one of the
conditions of approval.
Brigitte C. Widemann, MD, Principal Investigator of the SPRINT
trial and Chief, NCI Pediatric Oncology Branch, said: "For children
with neurofibromatosis type 1, plexiform neurofibromas can grow and
develop so significantly that, in some cases, it becomes
debilitating. In the SPRINT trial, selumetinib shrank
NF1-associated PNs in 66% of patients and showed clinically
meaningful improvements in PN-related symptoms."
Dave
Fredrickson, Executive Vice President, Oncology Business Unit,
said: "As the first medicine approved in the EU for patients with
neurofibromatosis type 1, Koselugo has the potential to transform
the way plexiform neurofibromas are managed and treated. The SPRINT
data showed that Koselugo
not only shrank tumours in some children, but also reduced pain and
improved their quality of life. This significant milestone was made
possible thanks to our research partners, the National Cancer
Institute, the Neurofibromatosis Therapeutic Acceleration Program,
the Children's Tumor Foundation, the patient community and every
child, parent and doctor involved in the clinical
trial."
Roy
Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories,
said: "Before this approval, surgery was the only treatment option
for children in the EU with neurofibromatosis type 1 plexiform
neurofibromas. This approval marks a significant step forward in
addressing the debilitating impact of these tumours."
The
SPRINT Stratum 1 Phase II trial showed Koselugo demonstrated an objective
response rate (ORR) of 66% (33 of 50 patients, confirmed partial
response) in paediatric patients with NF1 PN when treated with
Koselugo as twice-daily
oral monotherapy.8 ORR is defined as
the percentage of patients with confirmed complete (disappearance
of PN) or partial response (at least 20% reduction in tumour
volume).8
Results were published in The
New England Journal of Medicine.7
Koselugo is approved
in the US and several other countries
for the treatment of paediatric patients with NF1 and symptomatic,
inoperable PN. Further regulatory submissions are underway.
Clinical trials of Koselugo in adult patients with NF1 PN, including an
alternative age-appropriate formulation for paediatric patients,
are scheduled to begin this year.
NF1
NF1 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). In 30-50% of people, tumours develop on
the nerve sheaths.1,3,9,10 These PN can
cause clinical issues such as pain, motor dysfunction, airway
dysfunction, bladder/bowel dysfunction and disfigurement, as well
as having the potential to transform into malignant peripheral
nerve sheath tumours.4-7,10 PN begin
developing during early childhood, with varying degrees of
severity, and can reduce life expectancy by eight to 15
years.3,6,11,12
SPRINT
The
SPRINT Stratum 1 Phase 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.7 This trial sponsored
by NCI CTEP was conducted under a Cooperative Research and
Development Agreement between NCI and AstraZeneca with additional
support from Neurofibromatosis Therapeutic Acceleration Program
(NTAP).
Koselugo
Koselugo (selumetinib) is an inhibitor of mitogen-activated
protein kinase kinases 1 and 2 (MEK1/2).8 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.13
Koselugo received US FDA Breakthrough Therapy Designation in
April 2019, Rare Pediatric Disease Designation in December 2019 and
US Orphan Drug Designation in February 2018. Further orphan
designations have been granted in the EU, Japan, Russia,
Switzerland, South Korea, Taiwan and Australia.
AstraZeneca and MSD strategic oncology collaboration
In July
2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US,
known as MSD outside the US and Canada, announced a global
strategic oncology collaboration to co-develop and
co-commercialise Lynparza, the world's first PARP
inhibitor, and Koselugo (selumetinib),
a mitogen-activated protein kinase (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
is leading a revolution in oncology with the ambition to provide
cures for cancer in every form, following the science to understand
cancer and all its complexities to discover, develop and deliver
life-changing medicines to patients.
The
Company's focus is on some of the most challenging cancers. It is
through persistent innovation that AstraZeneca has built one of the
most diverse portfolios and pipelines in the industry, with the
potential to catalyse changes in the practice of medicine and
transform the patient experience.
AstraZeneca
has the vision to redefine cancer care and, one day, eliminate
cancer as a cause of death.
AstraZeneca
AstraZeneca
(LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical
company that focuses on the discovery, development and
commercialisation of prescription medicines in Oncology and
BioPharmaceuticals, including 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.
1.
Cancer.Net.
Neurofibromatosis Type 1. Available at: https://www.cancer.net/cancer-types/neurofibromatosis-type
1. Accessed June 2021.
2.
National Human
Genome Research Institute. About Neurofibromatosis. Available at:
https://www.genome.gov/Genetic-Disorders/Neurofibromatosis.
Accessed June 2021.
3.
Hirbe AC, Gutmann
DH. Neurofibromatosis type 1: a multidisciplinary approach to care.
Lancet Neurol.
2014;13:834-43. doi: 10.1016/S1474-4422(14)70063-8.
4.
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.
5.
Mayo Clinic.
Neurofibromatosis. Available at: https://www.mayoclinic.org/diseases-conditions/neurofibromatosis/symptoms-causes/syc-20350490.
Accessed June 2021.
6.
NHS.
Neurofibromatosis Type 1, Symptoms. Available at https://www.nhs.uk/conditions/neurofibromatosis-type
1/symptoms. Accessed June 2021.
7.
Gross AM, et al.
Selumetinib in Children with Inoperable Plexiform Neurofibromas.
N Engl J Med. 2020 Apr
9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735.
8.
European Medicines
Agency. Koselugo summary of
product characteristics. Accessed June 2021.
9.
Jett K, Friedman
JM. Clinical and genetic aspects of neurofibromatosis 1.
Genet Med. 2010:12(1):1-11.
doi: 10.1097/GIM.0b013e3181bf15e3. PMID: 20027112.
10.
Ghalayani P, Saberi
Z, Sardari, F. Neurofibromatosis Type I (von Recklinghausen's
Disease): A Family Case Report and Literature Review. Dent Res J.
2012;9(4):483-488.
11.
Evans DGR, Ingham
SL. Reduced Life Expectancy Seen in Hereditary Diseases Which
Predispose to Early Onset Tumors. Appl Clin Genet.
2013;6:53-61.
12.
NIH National
Institute of Neurological Disorders and Stroke. Neurofibromatosis
Fact Sheet. Available at: https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet.
Accessed June 2021.
13.
Koselugo (selumetinib) [prescribing
information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP;
2020.
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:
22 June
2021
|
By: /s/
Adrian Kemp
|
|
Name:
Adrian Kemp
|
|
Title:
Company Secretary
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