FDA approval of ULTOMIRIS based on data from two global, single-arm open-label studies of ULTOMIRIS, separately in adults and in children
Image: FDA Center for Drug Evaluation and Research. Photo: Courtesy of the U.S. Food and Drug Administration/Wikipedia.
US-based biopharmaceutical company Alexion has received the US Food and Drug Administration (FDA) approval for its ULTOMIRIS (ravulizumab-cwvz) for the treatment of atypical hemolytic uremic syndrome (aHUS).
ULTOMIRIS is a monoclonal antibody prescription medicine designed to treat aHUS by inhibiting complement-mediated thrombotic microangiopathy (TMA) in adults and infants aged one month and older.
Alexion research and development head and executive vice president John Orloff 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.”
FDA approval marks the first regulatory approval of ULTOMIRIS for paediatrics
Atypical HUS is a rare disease that causes continuous damage to the walls of blood vessels and blood clots in both children and adults, resulting in injury to vital organs, predominantly the kidneys.
The disease can induce potentially irreversible damage to kidneys and other vital organs, sudden or progressive kidney failure, requiring supportive care, including dialysis, in an intensive care unit or transplantation, and premature death in some cases.
Alexion said that the FDA has approved the drug based on data from two global, single-arm open-label studies of ULTOMIRIS, separately in adults and in children (paediatrics).
Upper respiratory tract infection, diarrhoea, nausea, vomiting, headache, hypertension and pyrexia include the most frequent adverse reactions in the studies.
In addition, serious meningococcal infections are observed in patients treated with ULTOMIRIS, for which specific risk-mitigation plans, including a REMS, have been established to minimize the risk for patients using ULTOMIRIS.
The Ohio State University College of Medicine Wexner Medical Center Clinical Internal Medicine professor Spero Cataland said: “The primary approach to treatment is to prevent the body from attacking itself, through the inhibition of uncontrolled complement activation, referred to as 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 six or seven infusions a year in adults—and that is important to consider for my patients.”