The European Commission has approved Alexion Pharmaceuticals' Ultomiris (ravulizumab) for the treatment of some adults with the ultra-rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).

The decision allows use of the drug – the first and only long-acting C5 complement inhibitor administered every eight weeks – in patients with haemolysis with clinical symptoms indicative of high disease activity, and also for those who are clinically stable after having been treated with Soliris (eculizumab) for at least the past six months.

PNH is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can affect men and women without warning, with an average age of onset in the early 30s.

In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, causes the destruction of red blood cells, which can lead to progressive anaemia, fatigue, dark urine, and shortness of breath.

In the most severe cases, the condition can cause the formation of blood clots, potentially damaging vital organs and causing premature death.

Data from a pivotal Phase III study showed that Ultomiris was non-inferior to Soliris (eculizumab) in complement inhibitor treatment-naïve patients with PNH, based on the co-primary endpoints of transfusion avoidance and normalisation of lactate dehydrogenase (LDH) levels.

"More than a decade after SOLIRIS was approved, Ultomiris provides a major step forward in the treatment of PNH," said Alexander Röth, Department of Haematology, West German Cancer Center, University Hospital Essen, Germany.

"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 Ohase III program conducted in PNH so far."