FDA Approves Ultomiris Therapy For Pediatric Patients With PNH

The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) therapy to treat pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH), a "rare, life-threatening blood disease."

With a median survival rate of only 10 years after diagnosis, PNH is characterized by blood clots, anemia, red blood cell destruction, and impaired bone marrow function.

Risks associated with taking Ultomiris range from fatal to minor and include Meningococcal infection, infusion-related reactions, upper respiratory tract infection and headache. Ultomiris injection therapy is available in an exclusive program under an approved risk evaluation and mitigation strategy to lessen morbidity risks and mitigate the frequency of infection, specifically Meningococcal infection.

Learn more on the FDA website.

Clinical Topics: Novel Agents

Keywords: ACC Advocacy, Hemoglobinuria, Paroxysmal, United States Food and Drug Administration, Antibodies, Monoclonal, Humanized, Hematologic Diseases, Anemia, Thrombosis, Meningococcal Infections, Respiratory Tract Infections, Erythrocytes


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