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European approval for Alexion's Ultomiris in rare blood disease

Published on 04/07/19 at 10:31am

Alexion has received marketing authorisation from the European Commission for its long-acting C5 complement inhibitor Ultomiris (ravulizumab) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) with haemolysis with high disease activity, as well as for adult patients who are clinically stable after having been treated with Soliris (eculizumab) for at least six months.

The decision was reached based on data drawn from two Phase 3 studies encompassing 440 participants. The findings from these trials demonstrated that, when administered every eight weeks, Ultomiris proved itself to be as effective as Soliris every two weeks on all 11 primary and secondary endpoints. The drug also showed a similar safety profile to Soliris.

PNH is an ultra-rare condition which eventually result in thrombosis, a blood clot in a vessel which slows of prevents the flow of blood through the body, in serious cases causing organ damage, heart attack, stroke, or even death.

“More than a decade after Soliris was approved, Ultomiris provides a major step forward in the treatment of PNH,” explained Dr Alexander Röth, Department of Hematology, West German Cancer Center at Germany’s University Hospital Essen. “Now PNH patients can experience greater freedom in their lives with significantly fewer infusions per year without any compromise on efficacy or safety. This has been impressively demonstrated in the largest Phase 3 programme conducted in PNH so far.”

Dr John Orloff, Executive Vice President and Head of Research & Development at Alexion, added: “At Alexion, our goal is to continue to improve the lives of people and families affected by PNH and other serious rare diseases. We believe Ultomiris will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the eight week dosing interval, and reducing the number of infusions per year from 26 with Soliris to only six or seven with Ultomiris. We are also particularly pleased by the positive data showing patients can successfully transition from Soliris to Ultomiris.”

Alexion’s drug secured approval with the FDA for adults with PNH in December last year, and with the Japanese Ministry Of Health, Labour And Welfare on 18 June this year.

Matt Fellows

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