FDA Approves New Treatment for Paroxysmal Nocturnal Hemoglobinuria

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

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. 

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