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June 7, 2021Rare Blood Disorder Indication for Ultomiris Expanded to Include Pediatric Patients
June 7, 2021 – The U.S. FDA has approved an expanded indication for Ultomiris® (ravulizumab-cwvz), manufactured by Alexion Pharmaceuticals, to treat paroxysmal nocturnal hemoglobinuria (PNH) in patients who are at least one month old. Ultomiris first received FDA approval in 2018 to treat PNH in adults.
PNH is a rare, acquired genetic disorder that causes anemia due to destruction of red blood cells, thrombosis (blood clots), and impaired bone marrow function (inability to make enough new blood cells). Though in most instances the disease is identified when an individual is 35-40 years of age, it can occur in pediatric patients. The median survival time after diagnosis is 10 years. However, some patients can survive for decades and experience only minor symptoms.
In a clinical study, the efficacy and safety of Ultomiris in pediatric patients diagnosed with PNH were found to be consistent with the drug’s established profile in clinical studies involving adults, as well as representative of the safety and efficacy observed in real-world clinical settings.
Recommended dosing is based on patient weight and requires a loading dose. After the loading dose, maintenance doses are administered once every four or eight weeks, based on weight. Ultomiris is given as an intravenous infusion, with the infusion rate and time varying according to the patient’s weight.
A black box warning cautions that meningococcal infections and sepsis have occurred in patients treated with Ultomiris and may rapidly become life-threatening or fatal if not recognized and treated early. The drug is available only through a Risk Evaluation and Mitigation Strategy (REMS) program.