Ultomiris FDA approval : Alexion Pharmaceuticals has bagged approval from the US Food and Drug Administration (FDA) for Ultomiris (ravulizumab-cwvz) for the treatment of atypical hemolytic uremic syndrome (aHUS) in adults and pediatric (one month of age and older) patients.

The approval is for the prevention of complement-mediated thrombotic microangiopathy (TMA) in such patient populations. It marks the first pediatric approval for Ultomiris.

Considered to be an ultra-rare disease, Atypical HUS can lead to progressive injury to vital organs, especially the kidneys, through damage to the walls of blood vessels and blood clots. Atypical HUS can result in sudden organ failure or a slow loss of function over time, which can possibly lead to the necessity for a transplant, and in certain cases, death.

Commenting on Ultomiris FDA approval, John Orloff – Executive Vice President and Head of Research and Development at Alexion Pharmaceuticals, said: “The consequences of uncontrolled complement activation, like organ failure and potentially death, create significant challenges and uncertainty for people and families facing aHUS.

“Based on the Phase 3 data, which demonstrated clinically meaningful benefits in people with aHUS, we believe Ultomiris has the potential to become the new standard of care for this devastating disease.”

Ultomiris FDA approval has been given on the data from a couple of global, single-arm open-label clinical trials – one in adults and the other in children with aHUS.

The pediatric clinical trial is in progress in which 14 out of 16 children were enrolled and included in the interim analysis. Efficacy evaluation of Complete TMA Response was defined by hematologic normalization parameters and enhanced kidney function.

According to Alexion Pharmaceuticals, 54% of adults and 71% (interim data) of children showed Complete TMA Response in the initial 26-week treatment periods.

Treatment with Ultomiris decreased thrombocytopenia (low blood platelet count) in 84% of adults and 93% of children, reduced hemolysis (the destruction of red blood cells) in 77% of adults and 86% of children, and enhanced kidney function in 59% of adults and 79% (interim data) of children.

Alexion Pharmaceuticals said that regulatory filings for marketing authorizations of Ultomiris for aHUS are under review in the European Union (EU) and Japan.

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  Alexion Pharmaceuticals, atypical hemolytic uremic syndrome, John Orloff, ravulizumab-cwvz, Ultomiris, US FDA, US Food and Drug Administration, USA