Study reveals positive impact of Soliris on long-term outcomes in PNH patients

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced the presentation of research that provides further insight into the clinical consequences of paroxysmal nocturnal hemoglobinuria (PNH), and positive impact of Soliris® (eculizumab) therapy on long-term outcomes. The data, presented this weekend at the 16th Congress of the European Hematology Association (EHA) in London, included studies on mortality in PNH patients with renal impairment, long-term outcomes with Soliris therapy, disease burden in non-transfused patients with PNH, and other areas of research in PNH and Soliris therapy.

“Clinical impact of uncontrolled complement activity in Japanese non-transfused patients with paroxysmal nocturnal hemoglobinuria”

PNH is an ultra-rare, life-threatening blood disorder in which uncontrolled activation of the complement system causes the chronic destruction of red blood cells (hemolysis). Soliris, a first-in-class terminal complement inhibitor, is the only therapy approved for the treatment of patients with PNH.

"Research presented at EHA further quantifies the severe impact of PNH and the proven clinical benefits of long-term Soliris therapy in these patients," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Through clinical trials, independent research, and international registries, physicians are gaining a more complete understanding of PNH and developing more effective strategies for diagnosing and treating patients."

Renal Impairment in PNH

In a poster presentation today, a study found that patients with PNH who have late-stage renal impairment had a significantly worse overall survival rate compared with PNH patients without renal impairment. Researchers analyzed data from 301 patients enrolled in the National Data Registry in South Korea to assess the impact of late-stage renal impairment in patients with PNH: 16% of patients had a history or presence of late-stage renal impairment, and these patients accounted for 35% of patient deaths in the registry. Patients with late-stage renal impairment had significantly worse overall survival compared with PNH patients with no impairment.

"Late-stage renal impairment is a common, severe and underappreciated consequence of PNH and a strong predictor of death in people with this disease," said Jin Seok Kim, Division of Hematology, Severance Hospital, Yonsei University College of Medicine, Seoul, Korea. "This study confirms the connection between renal impairment and PNH, and provides a strong rationale for the effective treatment of hemolysis, the underlying cause of organ damage in patients with PNH."

Long-Term Outcomes with Soliris Therapy

In a poster session held yesterday, researchers presented long-term data from all 195 patients who participated in the Soliris PNH clinical trials and extension studies. The study showed that, among these patients, overall survival with Soliris therapy was 97.6% at three years and was maintained through 5.5 years. These results are consistent with data published in the journal Blood earlier this year, which demonstrated that survival of studied patients with PNH who were treated with Soliris was no different than survival in an age- and gender-matched normal population. The current study also showed a reduction of thromboembolic events (TE) from 52 pre-treatment events to 10 events during Soliris therapy, in a time-matched analysis, and a reduction in the prevalence of chronic kidney disease from 69% of patients at baseline to 31% after 36 months of treatment. These results were previously presented at the 52nd American Society of Hematology Annual Meeting.

"This analysis suggests that chronic complement inhibition with Soliris can significantly improve the poor prognosis for patients with PNH," said Peter Hillmen, M.D., Ph.D., consultant haematologist at the Leeds Teaching Hospitals NHS Trust and lead author of the study. "In this analysis, patients who received Soliris treatment for more than five years were significantly less likely to experience life-threatening outcomes such as chronic kidney disease or thromboembolism."

Outcomes in Non-Transfused Patients

In another study presented at EHA, investigators showed that patients with no history of transfusion demonstrated substantial disease burden despite their transfusion status. A poster presentation today reported on two patients from the AEGIS study with no history of transfusion. Both patients showed evidence of significant clinical disease burden at baseline, including chronic hemolysis and renal disease. In both patients, Soliris treatment resulted in substantial reductions in LDH (a measure of hemolysis), clinically meaningful improvement in fatigue and quality of life, and reduction of CKD.

New Data From International PNH Registry

A poster presented today compared the clinical characteristics of pediatric and adult patients enrolled in the International PNH Registry. The two groups showed similarities in LDH levels, hemoglobinuria, abdominal pain, underlying bone marrow disorders, history of renal impairment and other PNH-related symptoms at enrollment. In addition, the study indicates that pediatric patients with PNH are at risk for thromboembolism events (TE)..


  1. Jay Frame Jay Frame United States says:

    More recently, Soliris has also been approved by the FDA to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.

    Soliris’ safety and effectiveness were established in two single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 pediatric patients and 11 adult patients with aHUS. Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients with aHUS that did not respond to plasma therapy. Patients treated with Soliris also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

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