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# Ultomiris OK’d for Pediatric Blood Disorder
- URL: https://www.fdaweb.com/ultomiris-okd-for-pediatric-blood-disorder/
- Published: 2021-06-07T12:00:00.000Z
- Updated: 2026-09-14T17:02:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149479

FDA has approved an Alexion Pharmaceuticals BLA for Ultomiris (ravulizumab-cwvz) injection and its use in treating patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH). The a rare, life-threatening blood disease is characterized by red blood cell destruction, anemia, blood clots, and impaired bone marrow function. Ultomiris was originally approved in 2018 and is also indicated to treat adults with PNH and adult and pediatric patients with atypical hemolytic uremic syndrome.

The approval was based on data from a 13-patient study. Five patients had never been treated with complement inhibitors and eight patients had been treated with Alexion’s PNH therapy eculizumab. “After the 26-week study, 60% of patients who had not previously received complement inhibitors avoided a transfusion and all patients who had received prior eculizumab treatment avoided a transfusion,” an [agency release](https://content.govdelivery.com/accounts/USFDA/bulletins/2e2f48e?ref=fdaweb.com) says.  

Ultomiris is available only through a restricted program. FDA says. Meningococcal infections and sepsis can occur in patients taking Ultomiris, which can become life-threatening or fatal if not recognized and treated early. Patients also should be monitored for infusion-related reactions., it says.