Shares in pharmaceutical giant AstraZeneca (AZN) gained 0.5% to £86.60 after its rare blood disease drug Ultomiris was approved by the EU for treating children and adolescents.
The approval by the European Commission was based on interim results from a phase three trial on young people with a bodyweight above 10 kilos which demonstrated the required efficacy and safety.
Ultomiris was developed to treat PNH (paroxysmal nocturnal haemoglobinuria) which 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.
The drug was approved in the EU in 2019 for the treatment of adults with PNH and in June 2021 it was approved by the US Food and Drug Administration for children and adolescents, the first and only treatment for this age group in the US.
RARE DISEASE SPECIALIST
The drug was developed by AstraZeneca’s rare drug specialist division Alexion which AstraZeneca purchased last year in a shares and cash transaction valued at $39 billion.
Alexion has been a leader in rare diseases for over 30 years and is focused on serving patients and families affected by rare diseases through the discovery, development, and commercialisation of life-changing medicines.
