FDA approves Alexion’s Ultomiris in atypical haemolytic uremic syndrome for adults and children

pharmafile | October 21, 2019 | News story | Sales and Marketing Alexion, FDA, Ultomiris, pharma, rare disease 

The FDA has moved to expand the existing label for Alexion’s Ultomiris (ravulizumab-cwvz) to include the treatment of atypical haemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy in patients aged one month and older.

Atypical HUS is an ultra-rare condition characterised by blood clots and progressive damage to walls of blood vessels, leading to loss of function or even sudden failure of vital organs, particularly the kidneys.

The decision from the US regulator follows data from two single-arm studies into both children and adult patients, demonstrating that, in in the initial 26-week treatment period, 54% of adult participants and 71% of paediatric participants achieved a complete Thrombotic Microangiopathy (TMA) Response, defined by haematologic normalisation and improvement in kidney function by at least 25%.

Treatment with Alexion’s drug also reduced thrombocytopenia in 84% and 93% of adults and children, reduced haemolysis in 77% and 86%, and improved kidney function in 59% and 79% respectively.

“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,” said Dr Spero Cataland, Professor of Clinical Internal Medicine, Wexner Medical Center at the Ohio State University College of Medicine. “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.”

Dr John Orloff, Executive Vice President and Head of Research and Development at Alexion, added: “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.”

Matt Fellows

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