AstraZeneca’s rare disease unit Alexion has gained a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) to expand the use of its PNH drug Ultomiris to include the treatment of children and adolescents.
PNH is a rare blood condition where red blood cells are destroyed and can result in blood clots and impaired bone marrow function. Often, the rare disease goes unrecognised, with delays in diagnosis ranging from between one to more than five years.
The positive recommendation is based on interim results from a phase 3 trial, which showed that Ultomiris (ravulizumab) was effective in achieving complete C5 complement inhibition for 26 weeks in children and adolescents up to 18 years of age.
The C5 complement inhibitor also had no reported treatment-related severe adverse events, with no patients discontinuing treatment during the primary evaluation period or experiencing breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.
Specifically, the CHMP has recommended the expanded use of Ultomiris to include children and adolescents with PNH who experience haemolysis with clinical symptoms indicative of high disease activity and for individuals who are clinically stable after treatment with Alexion’s older PNH drug Soliris (eculizumab).
“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.
In July, AZ completed its acquisition of Alexion, establishing the company as its specialist rare disease unit.
Subsequently, AZ immediately gained assets within Alexion’s commercial portfolio, estimated to be worth around $6bn. This includes the company’s complement component 5 (C5) inhibitors Soliris and Ultomiris, which are available in the US and EU.
AZ also gained a portfolio of assets currently in clinical development, consisting of a pipeline of 11 molecules in over 20 clinical development programmes.