Credit: Globovision
The US Food and Drug Administration (FDA) has granted full approval for eculizumab (Soliris) to treat adult and pediatric patients with atypical
hemolytic uremic syndrome (aHUS).
The drug received accelerated approval for this indication in 2011.
Now, eculizumab has received full FDA approval based on the fulfillment of post-marketing requirements, including the submission of data from 2 additional prospective trials of eculizumab in patients with aHUS.
The revised eculizumab label now includes results with 2 years of ongoing treatment in aHUS patients and data on the use of eculizumab prior to supportive care with plasma or plasma exchange in prospective clinical trials.
The drug’s label also includes a boxed warning informing readers that life-threatening and fatal meningococcal infections have occurred in patients treated with eculizumab.
About aHUS and eculizumab
aHUS is a chronic, life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes chronic, uncontrolled complement activation. This results 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 and life-threatening damage to the kidney, brain, heart, and other vital organs, as well as premature death. Complement-mediated TMA also causes thrombocytopenia and hemolysis.
Eculizumab is a first-in-class terminal complement inhibitor indicated to inhibit complement-mediated TMA. The drug’s effectiveness in aHUS is based on its effects on TMA and renal function.
Eculizumab received accelerated FDA approval to treat aHUS in September 2011. The FDA granted this approval based on the results of 2 trials that suggested the drug was likely to provide a clinical benefit.
To achieve traditional FDA approval, the drug’s developer, Alexion Pharmaceuticals, was required to submit additional data that confirm the drug provides a clinical benefit.
To that end, the Clinical Studies section (Section 14.2) of the revised eculizumab prescribing information now contains results from 4 prospective, single-arm studies in patients with aHUS.
This includes updated data from the first 2 trials, as well as data from 2 new trials, 1 in pediatric patients with aHUS and the other in adolescents and adults with aHUS.
For details on these trials, see the full prescribing information.
