Alexion Pharmaceuticals’ Ultomiris (ravulizumab-cwvz) has been found to be successful in treating atypical haemolytic uremic syndrome (aHUS) in a Phase III study.

In the initial 26-week treatment period, 53.6% of patients demonstrated complete thrombotic microangiopathy (TMA) response, and the drug provided immediate and complete inhibition of the complement C5 protein that was sustained over the entire eight-week dosing interval.

Treatment with Ultomiris also reduced thrombocytopaenia, as measured by normalisation in platelet count, in 83% of patients, and reduced haemolysis (the destruction of red blood cells), as measured by normalisation in lactate dehydrogenase (LDH) level, in 76.8% of patients.

“We are very pleased with these data, which demonstrate that Ultomiris can provide clinically meaningful benefits to patients with aHUS” said John Orloff, executive vice president and head of research & development at Alexion.

“The results met the high bar of complete TMA response, defined by haematologic normalisation and improved kidney function, and provide confidence that Ultomiris has the potential to become the new standard of care for patients with aHUS."

The firm said it is preparing regulatory submissions for Ultomiris in aHUS in the US, EU and Japan "as quickly as possible.”

aHUS is a severe and chronic ultra-rare disease that can cause progressive damage to vital organs, predominantly the kidneys, leading to kidney failure and premature death.