Soliris was well tolerated in the study. The most common drug-related AEs were headache, leukopenia, lymphopenia, and cough/productive cough. 2
Efficacy of Soliris Regardless of Identified Genetic Mutation
In a poster session on November 1, researchers presented an analysis of efficacy parameters across three Soliris studies (two prospective and one retrospective) based on the presence or absence of genetic abnormalities.
An estimated 50-70% of patients with aHUS have identifiable genetic mutations or complement factor H (CFH) auto-antibodies that cause chronic, uncontrolled, and excessive activation of the complement system.
The risk of death or end-stage renal disease (ESRD) is similar in patients both with and without an identified mutation.
Across the three Soliris studies, the efficacy of Soliris – as measured by TMA event-free status, platelet normalization, hematological normalization, and improvement in measures of renal function – was similar in patients regardless of the presence or absence of an identified genetic abnormality.
“In three separate clinical studies, aHUS patients with and without identified genetic mutations or CFH auto-antibodies demonstrated similar improvements in clinical outcomes, including reduced disease burden, reduced complement-mediated TMA, improved renal function, and reduced need for supportive care intervention,” said Timothy Goodship, M.D., Professor of Renal Medicine at Newcastle University and the lead author of the poster. “Given the risk of life-threatening systemic TMA events and organ damage in these patients, and the greater clinical improvements associated with early treatment, Soliris therapy can be started immediately at the time of diagnosis, without the delay of waiting for genetic testing, which can take several months to complete.”
aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.
Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.
Sixty-five percent of all patients with aHUS die, require kidney dialysis, or have permanent kidney damage within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI).
The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate in these TMA patients.
aHUS affects both children and adults.
Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the US, European Union, Japan and other countries as the first and only treatment in patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, chronic, ultra-rare, and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is indicated to reduce hemolysis.