FDA Approves Ultomiri® for Rare Disease aHUS

The U.S. Food and Drug Administration (FDA) approved Ultomiris® (ravulizumab) for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) for adult and pediatric (aged one month or older) patients.

aHUS is an ultra-rare blood disease that can cause progressive injury to vital organs.

Improved outcomes observed for adults and children

The approval was based on data from two global, single-arm, open-label studies—one in adults and the other in children. The pediatric study is ongoing; 14 of 16 children were enrolled and included in the interim analysis. Ravulizumab is administered intravenously every eight weeks (or every four weeks for pediatric patients weighing less than 20 kg), following a loading dose.

Efficacy evaluation of Complete TMA Response was defined by hematologic normalization parameters and improved kidney function (as measured by ≥25% improvement in serum creatinine from baseline).

In the initial 26-week treatment periods, 54% of adults and 71% of children demonstrated Complete TMA Response. Ravulizumab also reduced thrombocytopenia in 84% of adults and 93% of children, reduced hemolysis in 77% of adults and 86% of children, and improved kidney function in 59% of adults and 79% of children.

The most common adverse events associated with ravulizumab include upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension, and pyrexia. Ravulizumab includes a Boxed Warning about the risk of life-threatening meningococcal infections and sepsis. Patients should be immunized with meningococcal vaccines at least two weeks prior to the first dose of ravulizumab, unless the risks of delaying treatment outweigh the risks of developing a meningococcal infection.

Ravulizumab was previous FDA-approved for adults with paroxysmal nocturnal hemoglobinuria. Soliris® (eculizumab) is also FDA-approved to treat aHUS in adults, but ravulizumab has a half-life that is three to four times longer.