# AstraZeneca’s Ultomiris approved in EU for children with PNH

- Link: https://www.directorstalkinterviews.com/astrazenecas-ultomiris-approved-in-eu-for-children-with-pnh/4121020642
- Published: 2021-09-03T07:21:57+01:00
- Author: Amilia Stone

**[AstraZeneca plc](https://www.directorstalkinterviews.com/ftse-company/astrazeneca/azn.l)**(
[LON:AZN](https://www.directorstalkinterviews.com/ftse-company/astrazeneca/azn.l))
_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.

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