Alexion Pharma (ALXN) Says Data from Global aHUS Registry Shows Reduced Risk with Soliris

November 17, 2016 5:33 PM EST

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Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that researchers presented new data from an analysis of patients enrolled in the Global atypical Hemolytic Uremic Syndrome (aHUS) Registry, demonstrating that initiation of Soliris® (eculizumab) prior to kidney transplant reduces the risk of dialysis post-transplant in patients with aHUS. Specifically, results showed that starting Soliris prior to transplant reduced the likelihood of dialysis three-fold compared to initiating Soliris post-transplant. There was also a four-fold reduction in chronic dialysis when Soliris was initiated prior to transplant and maintained in accordance with labeled dosing. Further, the need for dialysis increased after transplant if Soliris was discontinued.1 These data were presented in an oral session at the 2016 annual meeting of the American Society of Nephrology (ASN) in Chicago.

“Patients suffering from the devastating effects of aHUS are at a high risk of graft loss following kidney transplant due to the ongoing, unpredictable risk of complement-mediated thrombotic microangiopathy,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “The data presented at ASN show that initiating Soliris prior to transplant has the potential to reduce the risk of both acute and chronic dialysis, and demonstrate the benefit of sustained treatment in patients with aHUS.”

aHUS is a genetic, chronic, and progressive ultra-rare disease associated with vital organ failure and premature death.2,3,4 Soliris is approved in nearly 40 countries as a treatment for patients with aHUS and in nearly 50 countries as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). Both aHUS and PNH are caused by chronic uncontrolled complement activation.

Researchers at ASN will also present an update on renal survival characteristics of patients enrolled in the Global aHUS Registry in a poster presentation at ASN on Saturday, November 19 (SA-PO783).5

Timing of Eculizumab Treatment and the Need for Dialysis in Patients with aHUS Who Receive a Kidney Transplant (TH-OR095)1

In an oral session, Andrew Siedlecki, M.D., Instructor in Medicine, Brigham and Women’s Hospital, Boston, presented results from an analysis of patients enrolled in the Global aHUS Registry evaluating the rate of dialysis in 147 patients with aHUS who had a kidney transplant and were treated with Soliris, and had at least 1 year of follow-up post-transplant. The study compared all patients who started on Soliris therapy before or at the time of the transplant (n=69, median age 34 at current transplant, including patients receiving non-labeled dosing) to those who started on Soliris after the transplant (n=78, median age 35). A sub-analysis was also performed on the patients who continued to receive labeled dosing of Soliris initiated prior to transplant (n=53) compared to those who were initiated on and continued to receive labeled dosing of Soliris after the transplant (n=65).

Dr. Siedlecki reported that:

  • Six out of 69 patients who started on Soliris therapy before transplant required dialysis, compared to 28 out of 78 who initiated Soliris treatment post-transplant. The adjusted hazard ratio was 3.0 (95% CI 1.2-7.7) comparing dialysis events in the two populations, indicating a three-fold increase in the likelihood of dialysis in the patients who initiated Soliris treatment post-transplant.
  • In the sub-analysis of patients receiving labeled dosing, 4 out of 53 patients who started Soliris therapy before transplant required dialysis, compared to 23 out of 65 patients who initiated Soliris treatment post-transplant, indicating a nearly three-fold increase in likelihood of dialysis in the patients initiating Soliris treatment post-transplant. In addition, 1 out of 53 patients who started on Soliris therapy before transplant required chronic dialysis, compared to 9 out of 65 patients who initiated Soliris treatment post-transplant, indicating a four-fold increase in the likelihood of chronic dialysis in patients initiating Soliris treatment post-transplant.

“The aim of this analysis was to evaluate the timing of eculizumab treatment and the need for dialysis in patients with aHUS who receive a kidney transplant,” said lead study investigator, Dr. Siedlecki. “These results suggest that initiating treatment with eculizumab prior to kidney transplant, and maintaining treatment, reduces the risk of initiating dialysis after transplant in patients with a clinical diagnosis of aHUS.”

The Global aHUS Registry is dedicated to increasing the knowledge of the natural history of aHUS, irrespective of management strategy, to help optimize care and improve quality of life for patients. Data from the registry serve to enhance the understanding of aHUS, as well as the use of Soliris as a treatment for patients with aHUS.



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