TIDMAZN
RNS Number : 5248T
AstraZeneca PLC
25 July 2022
25 July 07:05 BST
Ultomiris recommended for approval in the EU by CHMP for the
treatment of adults with generalised myasthenia gravis
First and only long-acting C5 complement inhibitor showed early
effect and demonstrated clinical improvement in activities of daily
living
Ultomiris (ravulizumab) has been recommended for marketing
authorisation in the European Union (EU) as an add-on to standard
therapy for the treatment of adult patients with generalised
myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR)
antibody-positive. If authorised, Ultomiris would be the first and
only approved long-acting C5 complement inhibitor for the treatment
of AChR antibody-positive gMG in the EU.
The Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency based its positive opinion on results
from the CHAMPION-MG Phase III trial.
In this trial, 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)
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 the EU is estimated
at approximately 89,000.(3-9)
Renato Mantegazza, Professor at the Department of
Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS
Istituto Neurologico Carlo Besta, Milan, Italy, and CHAMPION-MG
trial investigator, said: "gMG is a rare and complex disorder that
requires early, consistent and reliable intervention. This positive
recommendation offers hope to the community for an effective
long-acting option that will require fewer treatments each year and
help improve disease management."
Marc Dunoyer, Chief Executive Officer, Alexion, said: "This
recommendation is a major milestone in our commitment to help a
broader range of gMG patients, including those with milder
symptoms, and expand access to Ultomiris. As we listen to the
patient community, we're focused on understanding and meeting the
needs of gMG patients, providing them with effective and accessible
treatment options, and the positive opinion is a great step
forward."
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 drug reactions are diarrhoea, upper respiratory tract
infection, nasopharyngitis and headache.(1)
The CHMP recommended approval as an add-on to standard therapy
for the treatment of adult patients with gMG who are AChR
antibody-positive.
Ultomiris was approved in the US for adults with gMG who are
AChR antibody-positive in April 2022, and regulatory reviews are
ongoing in additional countries, including Japan.
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.(2,4,5,10,11) 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.(12-14)
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.(15,16)
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.(17)
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 for the treatment of certain
adults with gMG.
Ultomiris is also approved in the US, EU and Japan for the
treatment of certain adults and children with PNH.
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 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-cwvz) US 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. Westerberg E, Punga AR. Epidemiology of Myasthenia Gravis in
Sweden 2006-2016. Brain Behav. 2020;10:e01819.
https://doi.org/10.1002/brb3.1819
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. Fang, F., Sveinsson O., Thormar G., Granqvist M., Askling J.,
Lundberg IE., Ye W., (2015). The autoimmune spectrum of myasthenia
gravis: a Swedish population-based study. J Intern Med 2015;
277:594-604.
7. Lefter, S., Hardiman, O., Ryan, A., (2017). A
population-based epidemiologic study of adult neuromuscular disease
in the Republic of Ireland. Neurology 2017;88:304-313.
8. Pallaver, F., Riviera, AP., Piffer, S., Ricciardi, R., Roni,
R., Orrico, D., Bonifati, DM., (2011). Change in Myasthenia Gravis
Epidemiology in Trento, Italy, after Twenty Years.
Neuroepidemiology 2011;36:282-287.
9. Santos, E., Coutinho, E., Moreira, I., et.al., (2016).
Epidemiology of Myasthenia Gravis in Northern Portugal: Frequency
Estimates and Clinical Epidemiological Distribution of Cases.
Muscle Nerve 2016; 54: 413-421.
10. 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.
11. 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.
12. Myasthenia Gravis. National Organization for Rare Disorders
(NORD). Available here . Accessed March 2022.
13. Howard, J. F., (2015). Clinical Overview of MG. Available
here . Accessed March 2022.
14. 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.
15. Myasthenia Gravis Fact Sheet. (2020, April 27). National
Institutes of Neurological Disorders and Stroke. Available here .
Accessed March 2022.
16. 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).
17. 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
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