a3696g
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 July 2021
Commission
File Number: 001-11960
AstraZeneca PLC
1
Francis Crick Avenue
Cambridge
Biomedical Campus
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United
Kingdom
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Ultomiris
recommended in EU for children with PNH
26 July 2021 07:00 BST
Ultomiris recommended for approval in the EU by CHMP
for
children and adolescents with paroxysmal nocturnal
haemoglobinuria
Opinion
based on results from Ultomiris Phase III trial
that showed an established efficacy and safety profile with reduced
treatment burden for children with PNH and their
families
Alexion's Ultomiris (ravulizumab) has been recommended for
marketing authorisation in the European Union (EU) for expanded use
to include children and adolescents with paroxysmal nocturnal
haemoglobinuria (PNH).
The Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency based its positive opinion on interim
results from the Phase
III clinical trial in
children and adolescents with PNH, which were recently presented
during the European Hematology Association 2021 Virtual
Congress.
This trial demonstrated that Ultomiris was
effective in achieving complete C5 complement inhibition through 26
weeks for the treatment of children and adolescents up to 18 years
of age with PNH. Additionally, Ultomiris had
no reported treatment-related severe adverse events, and no
patients discontinued treatment during the primary evaluation
period or experienced breakthrough haemolysis, which can lead to
disabling or potentially fatal blood clots.1
The efficacy and safety of Ultomiris in
children and adolescents is consistent with the established profile
of Ultomiris in
clinical trials involving adults with PNH and is representative of
the broad PNH patient population seen in the real-world clinical
setting.2,3
PNH is an ultra-rare and severe blood disorder characterised by the
destruction of red blood cells that can cause a wide range of
debilitating symptoms and complications, including thrombosis
(blood clots), which can occur throughout the body, and result in
organ damage and potentially premature death.1,4-6
Austin Kulasekararaj, MD, King's College Hospital, London, United
Kingdom, said: "PNH can have a significant and devastating impact
on a child's quality of life, and it can be overwhelming for
families to manage their disease. The potential approval
of Ultomiris, which offers the efficacy and safety already
established with Soliris (eculizumab) and requires fewer treatments
each year, would have a meaningful impact for children with PNH and
their families."
Marc Dunoyer, incoming Chief Executive Officer, Alexion, said:
"This recommendation shows that Ultomiris - which has become the standard of care for
the treatment of adults with PNH - has the potential to transform
the lives of children and adolescents in Europe suffering from this
devastating rare disease. As we listen to the patient
community and understand the challenges of living with a rare
disease, we recognize the importance of continuing to deliver
options and formulations that enhance patient care and disease
management."
The CHMP recommended the expanded use of Ultomiris to
include children (with a body weight of 10 kg or above) and
adolescents with PNH who experience haemolysis with clinical
symptom(s) indicative of high disease activity, as well as for
individuals who are clinically stable after having been treated
with Soliris for at least the past six
months.
Ultomiris was first
approved in the EU in 2019 for the treatment of adults with PNH and
is also approved in the EU for the treatment of adults and children
with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the
US Food and Drug Administration approved the expanded use
of Ultomiris to
include children (one month of age and older) and adolescents with
PNH, the first and only treatment for this age group in the
US.
PNH
Paroxysmal nocturnal haemoglobinuria (PNH) is a serious ultra-rare blood disorder with
devastating consequences. It is characterised by the destruction of
red blood cells, which is also referred to as haemolysis. PNH
occurs when the complement system - a part of the body's immune
system - over-responds, leading the body to attack its own red
blood cells. PNH often goes unrecognised, with delays in diagnosis
from one to more than five years. Patients with PNH may experience
a range of symptoms, such as fatigue, difficulty swallowing,
shortness of breath, abdominal pain, erectile dysfunction,
dark-coloured urine and anaemia. The most devastating consequence
of chronic haemolysis is the formation of blood clots, which can
occur in blood vessels throughout the body, damage vital organs,
and potentially lead to premature death. The prognosis of PNH can
be poor in many cases, so a timely and accurate diagnosis - in
addition to appropriate treatment - is critical to improving
patient outcomes.
Ultomiris
Ultomiris (ravulizumab),
the first and only long-acting C5 complement inhibitor, offers
immediate, complete, and sustained complement inhibition. The
medication works by inhibiting the C5 protein in the terminal
complement cascade, a part of the body's immune system. When
activated in an uncontrolled manner, the complement cascade
over-responds, leading the body to attack its own healthy
cells. Ultomiris is
administered intravenously every eight weeks or, for paediatric
patients less than 20 kg, every four weeks, following a loading
dose. Ultomiris is
approved in the US for the treatment of adults and children (one
month of age and older) with PNH, as well as in the EU and Japan as
a treatment for adults with PNH. It is also approved in the US for
aHUS to inhibit complement-mediated thrombotic microangiopathy in
adult and paediatric (one month of age and older) patients, in the
EU for the treatment of adults and children with a body weight of
at least 10 kg with aHUS, as well as in Japan for adults and
children with aHUS.
Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca
focused on rare diseases, created following the 2021 acquisition of
Alexion Pharmaceuticals, Inc. As a leader in rare diseases for
nearly 30 years, Alexion is focused on serving patients and
families affected by rare diseases and devastating conditions
through the discovery, development and commercialisation of
life-changing medicines. Alexion focuses its research efforts on
novel molecules and targets in the complement cascade and its
development efforts on haematology, nephrology, neurology,
metabolic disorders, cardiology, ophthalmology, and acute care.
Headquartered in Boston, Massachusetts, Alexion has offices around
the globe and serves patients in more than 50
countries.
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, Rare Diseases, 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.
References
1.
Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood.
2014;124(18):2804-2811.
2.
Brodsky RA. Blood. 2008 Feb 15;111(4):1840-7.
3.
Hillmen P. N Engl J Med 2006 Sep 21;355(12):1233-43.
4.
Griffin M, Hillmen P, Munir T, et al. Significant hemolysis is not
required for thrombosis in paroxysmal nocturnal hemoglobinuria.
Haematologica. 2019;104(3):e94-e96.
5.
Holguin MH, Fredrick LR, Bernshaw NJ, Wilcox LA, Parker CJ.
Isolation and characterization of a membrane protein from normal
human erythrocytes that inhibits reactive lysis of the erythrocytes
of paroxysmal nocturnal hemoglobinuria. The Journal of clinical
investigation. 1989;84(1):7-17.
6.
Jang JH, Kim JS, Yoon SS, et al. Predictive factors of mortality in
population of patients with paroxysmal nocturnal hemoglobinuria
(PNH): results from a Korean PNH registry. J Korean Med Sci.
2016;31(2):214-221.
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:
26 July
2021
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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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