CHMP positive for Ultomiris to treat paroxysmal nocturnal haemoglobinuria

Alexion Pharmaceuticals, Inc. announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorization for Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor administered every eight weeks. The recommended indication is for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) with haemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with Soliris (eculizumab) for at least the past six months.

PNH is a severe, complement-mediated ultra-rare disease that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body and result in organ damage and premature death. The CHMP opinion is based on comprehensive results from two Phase III studies, which represent the largest Phase III program ever conducted in PNH.

In these two studies, which included more than 440 patients who had either never been treated with a complement inhibitor before, or who had been stable on Soliris, the efficacy of Ultomiris administered every eight weeks was non-inferior to the efficacy of Soliris administered every two weeks on all 11 endpoints. The safety profile of Ultomiris was similar to that of Soliris. Additional data showed that Ultomiris provided immediate and complete C5 inhibition that was sustained for eight weeks, and that Ultomiris eliminated breakthrough hemolysis associated with incomplete C5 inhibition. The entire clinical development program for Ultomiris in PNH to date represents more than 750 patient years of experience.

Comment: Ultomiris has the potential to become the new standard of care for both complement inhibitor-naïve patients and patients switching from Soliris (eculizumab) .

Comment; Ultomiris was FDA approved for PNH on 21 December 2018.