FDA approves Ultomiris for children with paroxysmal nocturnal hemoglobinuria
Click Here to Manage Email Alerts
The FDA approved ravulizumab-cwvz injection to treat children aged 1 month and older with paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria — a rare, life-threatening blood disease that affects one to 1.5 people per million, with 10% of cases occurring among children — is characterized by red blood cell destruction, anemia, blood clotting and impaired bone marrow function. Median survival is 10 years after diagnosis.
Researchers evaluated the efficacy of ravulizumab-cwvz (Ultomiris, Alexion) — previously approved for adults — among children in a 26-week study that enrolled 13 patients aged 9 to 17 years, eight of whom previously had received eculizumab (Soliris, Alexion).
Patients received a first dose of ravulizumab-cwvz followed by maintenance treatment 15 days later, with treatment continuing every 8 weeks for patients weighing at least 44 pounds and every 4 weeks for those weighing less than 44 pounds.
Pharmacokinetic and pharmacodynamic effects of treatment served as the study’s primary endpoints.
Sixty percent of patients who had not previously received complement inhibitors avoided a blood transfusion, a secondary endpoint, through week 26. All patients who previously received eculizumab avoided a transfusion.
Ravulizumab-cwvz is only available through a risk evaluation and mitigation strategy due to the risk for meningococcal infections and other infections.
Read more about
Click Here to Manage Email Alerts