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 September 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
approved
in EU for children with PNH
03 September 2021 07:00
BST
Ultomiris approved in the EU for children and
adolescents with paroxysmal nocturnal
haemoglobinuria
Approval based on interim results from Phase III trial
demonstrating complete terminal complement inhibition with reduced
dosing frequency compared to Soliris
Ultomiris (ravulizumab)
has been approved in the European Union for expanded use to include
children (with a body weight of 10 kg or above) and adolescents
with paroxysmal nocturnal haemoglobinuria
(PNH), an ultra-rare and
severe blood disorder characterised by the destruction of red blood
cells that can cause thrombosis (blood clots) and result in organ
damage and potentially premature death. 1-4
The approval by the European Commission was based on
interim results from the Phase III clinical trial in children and
adolescents with PNH that demonstrated the safety and efficacy
of Ultomiris in these patients.
Ultomiris has an established
safety and efficacy profile, offering reduced dosing frequency
compared to Soliris (eculizumab),
which is the current standard of care in the EU for the treatment
of children and adolescents with PNH. Ultomiris is administered
every four or eight weeks (depending on body weight), following a
loading dose. The approval
follows the recommendation of
the Committee for Medicinal Products for Human Use of the European
Medicines Agency in July 2021.
Austin Kulasekararaj, MD, King's College Hospital, London, UK,
said: "PNH is a devastating disease, and Ultomiris provides an advancement for paediatric
patients in the EU with an established safety and efficacy profile.
By requiring fewer infusions each year
than Soliris,
Ultomiris may reduce the
need for these young patients to miss school to receive
treatment."
Marc Dunoyer, Chief Executive Officer, Alexion, said: "This
approval of Ultomiris reflects our ongoing commitment to
delivering new treatments that can make a meaningful difference in
patients' lives. Ultomiris has become the standard of care for the
treatment of adults with PNH and we will make it available to this younger patient population as
soon as possible."
Interim results from the Phase III trial
demonstrated Ultomiris was effective in achieving complete C5
complement inhibition through 26 weeks for the treatment of
patients 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 profile of Ultomiris in children and adolescents is consistent
with the 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.5,6
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
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; in the EU for adults, as well as
for 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; and in
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 and ophthalmology. 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. 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.
3.
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.
4.
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.
5.
Brodsky RA. Blood. 2008 Feb 15;111(4):1840-7.
6.
Hillmen P. N Engl J Med 2006 Sep 21;355(12):1233-43.
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:
03 September 2021
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By: /s/
Adrian Kemp
|
|
Name:
Adrian Kemp
|
|
Title:
Company Secretary
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