FDA Approves Therapy for Pediatric Patients with Serious Rare Blood Disease FDA has approved Ultomiris (ravulizumab-cwvz) injection to treat patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. PNH is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells). The disease affects 1-1.5 people per million. Approximately 10% of cases are among pediatric patients. PNH can be serious, with median survival of 10 years after diagnosis. However, some patients live for decades with only minor symptoms. Ultomiris is available only through a restricted program under a risk evaluation and mitigation strategy. Meningococcal (a type of bacteria) infections/sepsis can occur in patients taking Ultomiris and can become life-threatening or fatal if not recognized and treated early. Patients with unresolved Neisseria Meningitidis infection or who are not vaccinated against this infection should not take Ultomiris unless the risks of delaying treatment outweigh the risks of developing a meningococcal infection. Other infections can occur as well. Patients also should be monitored for infusion-related reactions. The effect of withdrawal of anticoagulant therapy during Ultomiris treatment has not been established. The most common side effects of Ultomiris for patients with PNH are upper respiratory tract infection and headache. | 
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