European Commission Approves Alexion's Ravulizumab for PNH

Alexion announced Wednesday that the European Commission approved ravulizumab (Ultomiris) for adults with paroxysmal nocturnal hemoglobinuria (PNH) with hemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with eculizumab (Soliris) for at least the past 6 months.

Ravulizumab, to be sold under the name Ultomiris, is Alexion's long-acting C5 complement inhibitor, and offers less frequent administration than eculizumab. It is already approved for PNH in the United States, and last month, Alexion said the FDA accepted ravulizumab for priority review for the treatment of atypical hemolytic uremic syndrome (aHUS), with a decision expected by October 2019.

“At Alexion, our goal is to continue to improve the lives of people and families affected by PNH and other serious rare diseases,” said John Orloff, MD, executive vice president and head of research and development at Alexion in a statement. “We believe Ultomiris will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the 8-week dosing interval, and reducing the number of infusions per year from 26 with Soliris to only 6 or 7 with Ultomiris. We are also particularly pleased by the positive data showing patients can successfully transition from Soliris to Ultomiris.”

The European Commission approval is based on results from 2 phase 3 studies for PNH, which can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, resulting in organ damage and premature death. The studies included 441 patients who either had never been treated with a complement inhibitor before or who had been stable on eculizumab. The company said the efficacy of ravulizumab administered every 8 weeks was noninferior to the efficacy of eculizumab administered every 2 weeks on all 11 endpoints. Safety profiles were similar.

With biosimilar competition to eculizumab advancing, Alexion hopes to transition most of the patients already receiving the older eculizumab onto the newer ravulizumab in the near future.