TIDMAZN
RNS Number : 3634G
AstraZeneca PLC
26 July 2021
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
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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
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