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 August 2022
Commission
File Number: 001-11960
AstraZeneca PLC
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Francis Crick Avenue
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Biomedical Campus
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United
Kingdom
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Ultomiris approved in Japan for gMG
25 August 2022 07:10 BST
Ultomiris approved
in Japan for the treatment of adults with generalised myasthenia
gravis
First and only long-acting C5 complement inhibitor offers early
onset and sustained efficacy, and has the potential to reduce
treatment burden with dosing every 8 weeks
Ultomiris (ravulizumab)
has been approved in Japan for the treatment of adult patients with
generalised myasthenia gravis (gMG) who are anti-acetylcholine
receptor (AChR) antibody-positive and whose symptoms are difficult
to control with high-dose intravenous immunoglobulin therapy (IVIg)
or plasmaphaeresis. Japan's Pharmaceuticals and Medical
Devices Agency also indicated that Ultomiris can be considered for patients who cannot
receive high-dose IVIg or plasmaphaeresis due to complications,
adverse reactions or other limiting factors.
The approval of the first and only long-acting C5 complement
inhibitor by the Japanese Ministry of Health, Labour and
Welfare (MHLW) was based on positive results from the CHAMPION-MG
Phase III trial, which showed Ultomiris was superior to placebo in the primary
endpoint of change from baseline in the Myasthenia
Gravis-Activities of Daily Living Profile (MG-ADL) total score at
Week 26, a patient-reported scale that assesses patients' abilities
to perform daily activities.1 Additionally,
in prolonged follow-up results from the open-label extension,
clinical benefit of Ultomiris was observed through 60
weeks.1
gMG is a rare, debilitating, chronic, autoimmune neuromuscular
disease that leads to a loss of muscle function and severe
weakness.2 The
diagnosed prevalence of gMG in Japan is estimated at approximately
22,000.3
Hiroyuki Murai, MD, PhD, Professor and Chairman, Department of
Neurology, School of
Medicine, International
University of Health and Welfare, Narita, Japan, said: "C5
inhibition is a proven approach to manage gMG. The approval
of Ultomiris is an important advance for the gMG
community in Japan, offering patients and physicians a new
long-acting C5 inhibitor that has demonstrated sustained
improvement in activities of daily living through 60 weeks, with
fewer infusions per year over current
treatment."
Marc Dunoyer, Chief Executive Officer, Alexion, said: "We are
pleased that Ultomiris is now approved in Japan for adults with
gMG, a disease that may impact their ability to work, meet
family obligations and live their lives fully. The approval speaks
to the strength and consistency of Ultomiris clinical data as demonstrated in the global
CHAMPION-MG Phase III trial. We look forward to bringing this
treatment option to people living with gMG in Japan as part of our
broader strategy to expand global access to our medicines."
In CHAMPION-MG, the safety profile of Ultomiris was comparable to placebo and consistent
with that observed in Phase III trials of Ultomiris in paroxysmal nocturnal haemoglobinuria
(PNH) and atypical haemolytic uraemic syndrome (aHUS). The most
common adverse reactions in patients
receiving Ultomiris were nausea, headache and
diarrhoea.1
Results from the CHAMPION-MG trial were
published online in NEJM
Evidence and presented at
the 2022 American Academy of Neurology Annual Meeting in
April.
Ultomiris was approved in the
US for adults with
anti-AChR antibody-positive gMG in April, and regulatory reviews
are ongoing in additional countries. It was recently recommended
for marketing authorisation in the European Union as an add-on to
standard therapy for the treatment of adult patients with gMG who
are anti-AChR antibody-positive.
Notes
gMG
gMG is a rare autoimmune disorder characterised by loss
of muscle function and severe muscle weakness.2
Eighty percent of people with gMG are AChR antibody-positive
meaning they produce specific antibodies (anti-AChR) that bind to
signal receptors at the neuromuscular junction (NMJ), the
connection point between nerve cells and the muscles they
control.2,4-7 This
binding activates the complement system, which is essential to the
body's defence against infection, causing the immune system to
attack the NMJ.2 This
leads to inflammation and a breakdown in communication between the
brain and the muscles.2
gMG can occur at any age, but it most commonly begins for women
before the age of 40 and for men after the age of
60.8-10 Initial
symptoms may include slurred speech, double vision, droopy eyelids
and lack of balance; these can often lead to more severe symptoms
as the disease progresses such as, impaired swallowing, choking,
extreme fatigue and respiratory failure.11,12
CHAMPION-MG
The global Phase III randomised, double-blind, placebo-controlled,
multicentre 26-week trial evaluated the safety and efficacy
of Ultomiris in adults with gMG. The trial enrolled 175
patients across North America, Europe, Asia-Pacific and Japan.
Participants were required to have a confirmed myasthenia gravis
diagnosis at least six months prior to the screening visit with a
positive serologic test for anti-AChR antibodies, MG-ADL total
score of at least 6 at trial entry and Myasthenia Gravis Foundation
of America Clinical Classification Class II to IV at screening.
Patients could stay on stable standard of care medicines, with a
few exceptions, for the duration of the randomised control
period.13
Patients were randomised 1:1 to receive Ultomiris or placebo for a total of 26 weeks. Patients
received a single weight-based loading dose on Day 1, followed by
regular weight-based maintenance dosing beginning on Day 15, every
eight weeks. The primary endpoint of change from baseline in the
MG-ADL total score at Week 26 was assessed along with multiple
secondary endpoints evaluating improvement in disease-related and
quality-of-life measures.
Patients who completed the randomised control period were eligible
to continue into an open-label extension period evaluating the
safety and efficacy of Ultomiris, which is ongoing.
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 in adult patients, following a loading
dose.
Ultomiris is approved in
the US and Japan for the treatment of certain adults with
gMG.
Ultomiris is also approved
in the US, EU and Japan for the treatment of certain adults with
PNH and for certain children with PNH in the US and
EU.
Additionally, Ultomiris is approved in the US, EU and Japan for
certain adults and children with aHUS to inhibit
complement-mediated thrombotic microangiopathy.
As part of a broad development programme, Ultomiris is being assessed for the treatment of
additional haematology and neurology
indications.
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. Ultomiris (ravulizumab)
Japanese prescribing information; 2022.
2. Howard,
J. F., (2017). Myasthenia gravis: the role of complement at the
neuromuscular junction. Annals of The New York Academy of Sciences,
1412(1), 113-128.
3. Lai,
CH., Tseng, HK., (2010). Nationwide Population-Based
Epidemiological Study of Myasthenia Gravis in Taiwan.
Neuroepidemiology. 2010 June;35:66-71. 7.
4. Anil,
R., Kumar, A., Alaparthi, S., Sharma, A., Nye, JL., Roy, B.,
O'Connor, KC., Nowak, R., (2020). Exploring outcomes and
characteristics of myasthenia gravis: Rationale, aims and design of
registry - The EXPLORE-MG registry. J Neurol Sci. 2020 Jul
15;414:116830.
5. Oh
SJ., (2009). Muscle-specific receptor tyrosine kinase antibody
positive myasthenia gravis current status. Journal of Clinical
Neurology. 2009b Jun 1;5(2):53-64.
6. Tomschik,
M., Hilger, E., Rath, J., Mayer, EM., Fahrner, M., Cetin, H.,
Löscher, W., Zimprich, F., (2020). Subgroup stratification and
outcome in recently diagnosed generalized myasthenia gravis.
Neurology. 2020 Sep 8;95(10):e1426-e1436.
7. Hendricks,
TM., Bhatti, MT., Hodge, D., Chen, J., (2019). Incidence,
Epidemiology, and Transformation of Ocular Myasthenia Gravis: A
Population-Based Study. Am J Ophthalmol. 2019
Sep;205:99-105.
8. Myasthenia
Gravis. National Organization for Rare Disorders (NORD).
Available here.
Accessed March 2022.
9. Howard,
J. F., (2015). Clinical Overview of MG.
Available here.
Accessed March 2022.
10. Sanders,
D. B., Raja, S. M., Guptill J. T., Hobson-Webb, L. D., Juel, V. C.,
& Massey, J. M., (2020). The Duke myasthenia gravis clinic
registry: I. Description and demographics. Muscle & Nerve,
63(2), 209-216.
11. Myasthenia
Gravis Fact Sheet. (2020, April 27). National Institutes of
Neurological Disorders and Stroke. Available here.
Accessed March 2022.
12. Ding,
J., Zhao, S., Ren, K., Dang, D., Li, H., Wu, F., Zhang, M., Li, Z.,
& Guo, J., (2020). Prediction of generalization of ocular
myasthenia gravis under immunosuppressive therapy in Northwest
China. BMC Neurology, 20(238).
13. ClinicalTrials.gov.
Safety and Efficacy Study of Ravulizumab in Adults With Generalized
Myasthenia Gravis. NCT Identifier: NCT03920293.
Available here.
Accessed March 2022.
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:
25 August 2022
|
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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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