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EC approves Ultomiris for children and adolescents with PNH

The European Commission (EC) approved Ultomiris for children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).

Red blood cells in vein

AstraZeneca’s Ultomiris (ravulizumab) has been approved for expanded use in the EU, its use now includes children with a body weight of 10kg 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 and result in organ damage and potentially premature death.

The approval by the European Commission (EC) was based on interim results from a Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients. It follows the recommendation of the Committee for Medicinal Products for Human Use (CMPH) of the European Medicines Agency (EMA) in July 2021. 

Ultomiris is the first and only long-acting C5 complement inhibitor, and offers immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade. 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 20kg, every four weeks, following a loading dose. Ultomiris provides a reduced dosing frequency compared to Soliris (eculizumab), which is the current standard of care (SOC) in the EU for the treatment of children and adolescents with PNH. 

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. In addition, 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.

According to AstraZeneca, the efficacy and safety profile of Ultomiris in children and adolescents is consistent with the profile of the medication in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.

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 (FDA) approved the expanded use of Ultomiris to include children, aged one month and older, and adolescents with PNH, the first and only treatment for this age group in the US.

“PNH is a devastating disease, and Ultomiris provides an advancement for paediatric patients in the EU with an established safety and efficacy profile,” commented Austin Kulasekararaj at King’s College Hospital, UK. “By requiring fewer infusions each year than Soliris, Ultomiris may reduce the need for these young patients to miss school to receive treatment.”

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