AstraZeneca’s (AZ) Ultomiris has received a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) to expand its use to include children and adults with paroxysmal nocturnal haemoglobinuria (PNH).
The CHMP recommended marketing authorisation for expanded use of Ultomiris (ravulizumab) based on interim results from a Phase III trial of the treatment in children and adolescents with PNH.
In this trial, Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age.
The efficacy and safety of the long-acting C5 complement inhibitor in children and adolescents is consistent with its established profile in clinical trials involving adult PNH patients.
The CHMP has recommend expanded use for Ultomiris to include children with a body weight of 10kg or above and adolescents with PNH who experience haemolysis with clinical symptoms indicative of high disease activity.
The CHMP also included individuals who are clinically stable after having received Soliris (eculizumab) treatment for at least the past six months in its recommendation.
PNH is an ultra-rare, severe blood disorder that is characterised by the destruction of red blood cells. This can cause a myriad of symptoms and complications, including blood clots which can occur throughout the body and result in organ damage as well as potentially premature death.
“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,” said Marc Dunoyer, incoming chief executive officer of Alexion.
“As we listen to the patient community and understand the challenges of living with a rare disease, we recognise the importance of continuing to deliver options and formulations that enhance patient care and disease management,” he added.