Ravulizumab injection for blood disorder

February 5, 2019 0 By FM

Ravulizumab (Ultomiris) injection secured approval from the US FDA for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Ravulizumab is a long-acting complement inhibitor that prevents haemolysis in the life-threatening blood disease.

The FDA approval was backed by a clinical trial of 246 treatment naïve patients, who were randomized to be treated with ravulizumab or eculizumab, the current standard of care for PNH.

The results of the trial demonstrated that ravulizumab had similar results to eculizumab. The patients did not receive a transfusion and had similar incidence of haemolyses measured by the normalization of lactate dehydrogenase (LDH) levels in patients’ blood.

In addition, ravulizumab was studied in a second clinical trial of 195 patients with PNH who were clinically stable after having been treated with eculizumab for at least the past six months. These patients were randomly selected to be treated with ravulizumab or to continue eculizumab. Ravulizumab again demonstrated similar effects to eculizumab based on several clinical measures including haemolysis and avoiding transfusion.

The FDA granted the approval of ravulizumab to Alexion Pharmaceuticals.