Ultomiris for Adults With Rare, Life-Threatening Blood Disease

Less Burdensome Therapy for Patients With Paroxysmal Nocturnal Hemoglobinuria

Alexion Pharmaceuticals has received FDA approval for ravulizumab (Ultomiris) injection for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare and potentially fatal blood disease.

PNH, which is most often diagnosed in young adulthood, is an acquired disorder that leads to hemolysis. Sudden and recurring episodes lead to the destruction of red blood cells and may be activated by infections or physical exertion. Severe anemia, profound fatigue, shortness of breath, continual episodes of dark-colored urine, kidney disease, and recurrent pain can occur during the episodes.

Ravulizumab is a long-acting complement inhibitor that prevents hemolysis. Its novel formulation will bring some relief to patients, who will require treatment once every eight weeks. Eculizumab, the only other current standard of care, involves treatment every two weeks.

Results from two trials of 246 patients and 197 patients, respectively, have demonstrated that ravulizumab has similar results to eculizumab; patients were able to avoid transfusion and had a similar incidence of hemolysis.

Common side effects are headache and upper respiratory infection. Health care providers are should use caution when administering ravulizumab to patients with any other systemic infection.

Ravulizumab carries a boxed warning about the risk of life-threatening meningococcal infections and sepsis. Providers should comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.

The FDA granted ravulizumab priority review and orphan drug designations. Currently, the drug is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).

Source: FDA, December 21, 2018