The U.S. Food and Drug Administration (FDA) has approved the expanded use of ravulizumab (brand name: ULTOMIRIS) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is a rare, life-threatening blood disorder characterized by hemolysis. Persistently low hemoglobin levels can result in severe fatigue and difficulty breathing. Additionally, individuals with PNH are susceptible to thrombotic events and many have some degree of bone marrow dysfunction.
The approval of the expanded use was largely based on positive interim phase 3 study results, which showed that ravulizumab was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. Additionally, the drug had no reported treatment-related severe adverse events.
To learn more about PNH and other rare blood disorders, visit checkrare.com/diseases/hematologic-disorders/.
