Recently Approved Drug Ultomiris Performs Well in Atypical Hemolytic Uremic Syndrome Trial

According to a story from, the biotechnology company Alexion Pharmaceuticals recently announced that its product Ultomiris, which was recently approved as a treatment for paroxysmal nocturnal hemoglobinuria, was also recently successful in a trial of 56 patients with the rare kidney disease atypical hemolytic uremic syndrome. Ultomiris is essentially a longer lasting version of Soliris, another drug from the company. Patients that were previously taking Soliris are gradually being switched over to the new drug.

About Atypical Hemolytic Uremic Syndrome

Atypical hemolytic uremic syndrome is a very rare, progressive, and potentially life threatening illness which is most characterized by the formation of blood clots in many small blood vessels within the body. In at least some cases, atypical hemolytic uremic syndrome is the result of mutations affecting proteins that regulate the complement system, a component of the immune system. The uncontrolled activity of this system is what allows for the disease symptoms to appear, which can include fatigue, swelling, diarrhea, vomiting, abdominal pain, confusion, seizure, coma, kidney failure, heart attack, and stroke. This disease may appear alongside other illnesses as well. Treatment for this disease may include kidney transplant, plasma exchange/infusion, the drug eculizumab, and dialysis. More effective treatments are greatly needed for this illness, as patients have poor outcomes and overall quality of life. To learn more about atypical hemolytic uremic syndrome, click here.

Patients taking Ultomiris must receive an infusion every eight weeks, which is a considerable improvement over Soliris, which requires infusions every two weeks.

Trial Results

The results of the trial indicate that Ultomiris has some promise for treating atypical hemolytic uremic syndrome. After 26 weeks, 54 percent of the patients saw a return of their platelet levels to normal, improved function for their kidneys, and less destruction of their red blood cells. 

Considering the limited effectiveness of many current therapies, Ultomiris has the potential to become a new standard approach for atypical hemolytic uremic syndrome. Alexion hopes to have the drug approved in this indication within the EU, the US, and Japan as soon as it can. The company is continuing to develop the drug, including a version that can be administered more easily.


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