The FDA approved intravenous ravulizumab-cwvz (Ultomiris) to treat pediatric patients one month of age with paroxysmal nocturnal hemoglobinuria (PNH).
Ravulizumab is a complement inhibitor originally approved in 2018 for adult PNH patients. The disorder, which is characterized by red blood cell destruction, anemia, blood clots, and impaired bone marrow function, affects roughly 1-1.5 people per million, and approximately 10% of cases are among pediatric patients.
This approval was based on a 26-week study of ravulizumab that enrolled 13 pediatric patients ages 9-17 years with PNH.
“Five of the 13 patients had never been treated with complement inhibitors and eight patients had been treated with eculizumab, another PNH therapy,” the FDA explained. “Patients received a first dose of [ravulizumab] and a maintenance treatment after 15 days. Then, patients weighing 20 kg (44 lbs) or more received treatment every eight weeks while patients weighing less than 20 kg received treatment every four weeks.”
At the end of the study, 60% of patients who had not previously received a complement inhibitor and 100% of patients previously treated with eculizumab did not require blood transfusion.
Ravulizumab’s label includes a Boxed Warning stating that patients taking the drug are at increased risk for potentially fatal meningococcal infections and sepsis. “Patients with unresolved Neisseria Meningitidis infection or who are not vaccinated against this infection should not take [ravulizumab] unless the risks of delaying treatment outweigh the risks of developing a meningococcal infection,” the FDA wrote. Due to this risk, ravulizumab is only available through a risk evaluation and mitigation strategy.
The FDA also warned that other infections can occur in patients taking the drug, and patients should also be monitored for infusion-related reactions. The most commonly reported side effects are upper respiratory tract infection and headache.
Ravulizumab is manufactured by Alexion Pharmaceuticals.
John McKenna, Associate Editor, BreakingMED™
Cat ID: 138
Topic ID: 85,138,730,118,138,192,725,925
