Ravulizumab-cwvz (Ultomiris, Alexion) received FDA approval for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) for adult and pediatric patients.

This is ravulizumab-cwvz’s first pediatric approval, according to Alexion.

Atypical aHUS, an ultra-rare disease, can cause progressive injury to vital organs, which can result in sudden organ failure or a slow loss of function over time. Some cases may necessitate a transplant and sometimes may lead to death. Both children and adults can be affected by the disease.

According to Spero Cataland, MD, professor of clinical internal medicine at Wexner Medicine Center, The Ohio State University College of Medicine, treatment typically requires preventing the body from attacking itself through the inhibition of uncontrolled complement activation, also called C5 inhibition.

“Clinical study results showed adult and pediatric patients had complete C5 inhibition following the first dose of Ultomiris. C5 inhibition was sustained over time with only 6 or 7 infusions a year in adults—and that is important to consider for my patients,” Dr Cataland said in a statement.

The approval is based on data from 2 studies of patients with aHUS, 1 in adults and 1 in children.

The ongoing pediatric study includes a total of 14 out of 16 children enrolled and included in the interim analysis. Efficacy evaluation of complete TMA response was defined hematologic normalization parameters (platelet count and LDH) and improved kidney function (as measured by ≤ 25% improvement in serum creatinine from baseline).

Over the initial 26-week treatment period, 54% of adults and 71% (interim data) of children demonstrated complete TMA response, according to the study. The findings showed that treatment with ravulizumab-cwvz resulted in 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% (interim data) of children.

The most commonly reported adverse effects in these studies were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension, and pyrexia.

“The consequences of uncontrolled complement activation, like organ failure and potentially death, create significant challenges and uncertainty for people and families facing aHUS,” John Orloff, MD, executive vice president and head of research and development at Alexion, said in a statement. “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.”

Ravulizumab-cwvz is administered intravenously every 8 weeks or every 4 weeks for pediatric patients less than 20 kg following a loading dose, according to Alexion.

Reference

Alexion Receives FDA Approval for Ultomiris (Ravulizumab-Cwvz) for Atypical Hemolytic Uremic Syndrome (AHUS) [news release]. Alexion’s website. https://news.alexion.com/press-release/product-news/alexion-receives-fda-approval-ultomiris-ravulizumab-cwvz-atypical-hemolyt. Accessed October 2019.