The U.S. Food and Drug Administration has approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.

“The approval of Ultomiris will change the way that patients with PNH are treated,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Prior to this approval, the only approved therapy for PNH required treatment every two weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every eight weeks, without compromising efficacy.”

PNH is a rare acquired disorder that leads to the rupture or destruction of red blood cells (hemolysis). Patients with PNH are missing a certain protein that normally protects red blood cells from being destroyed by the patient’s immune system. Patients with PNH have sudden, recurring episodes where red blood cells are prematurely destroyed which may be triggered by stresses on the body, such as infections or physical exertion. During these episodes, the following symptoms may occur: severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark colored urine, kidney disease or recurrent pain. PNH can occur at any age, although it is most often diagnosed in young adulthood.

Ultomiris is a long-acting complement inhibitor that prevents hemolysis. The efficacy of Ultomiris was studied in a clinical trial of 246 patients who previously had not been treated for PNH (treatment naïve), who were randomized to be treated with Ultomiris or eculizumab, the current standard of care for PNH. The results of the trial demonstrated that Ultomiris had similar results to eculizumab (non-inferior) – patients did not receive a transfusion and had similar incidence of hemolysis measured by the normalization of LDH levels in patients’ blood (lactate dehydrogenase, or LDH, is an enzyme required during the process of turning sugar into energy in the body’s cells). In addition, Ultomiris 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 Ultomiris or to continue eculizumab. Ultomiris again demonstrated similar effects to eculizumab (non-inferior) based on several clinical measures including hemolysis and avoiding transfusion.

Common side effects reported by patients in clinical trials were headache and upper respiratory infection. Health care providers are advised to use caution when administering Ultomiris to patients with any other systemic infection.

The prescribing information for Ultomiris includes a Boxed Warning to advise health care professionals and patients about the risk of life-threatening meningococcal infections and sepsis. Health care providers are advised to comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Patients should be immunized with meningococcal vaccines at least two weeks prior to administering the first dose of Ultomiris, unless the risks of delaying treatment outweigh the risks of developing a meningococcal infection.

Patients and health care providers are advised that vaccination reduces, but does not eliminate, the risk of meningococcal infection. Patients should be monitored for early signs of meningococcal infections and evaluated immediately if infection is suspected. Ultomiris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Ultomiris must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

The FDA granted this application Priority Review designation. Ultomiris also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Ultomiris to Alexion Pharmaceuticals.

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