Additional Studies Presented at EHA Congress Describe Long-Term Efficacy and Survival Data with Sustained Soliris® (eculizumab) Therapy
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
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
"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
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. (2) 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
"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
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.(3) 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. (4) 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)..
About PNH
PNH is an ultra-rare blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in hemolysis (destruction of the patient's red blood cells). PNH strikes people of all ages, with an average age of onset in the early 30s. (5) Approximately 10 percent of all patients first develop symptoms at 21 years of age or younger. (6) PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years. (7) In the period of time before Soliris was available, it had been estimated that approximately one-third of patients with PNH did not survive more than five years from the time of diagnosis. (7) PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS). (8, 9, 10) In patients with thrombosis of unknown origin, PNH may be an underlying cause. (5) More information on PNH is available at www.pnhsource.com.
About Soliris
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris has been approved in the U.S.,
Important Safety Information
Soliris is generally well tolerated in patients with PNH. The most frequent adverse events observed in clinical studies of patients with PNH were headache, nasopharyngitis (runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.
The U.S. product label for Soliris also includes a boxed warning: "Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Vaccinate patients with a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary." During PNH clinical studies, two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. Prior to beginning Soliris therapy, all patients and their prescribing physicians are encouraged to enroll in the PNH Registry, which is part of a special risk-management program that involves initial and continuing education and long-term monitoring for detection of new safety findings.
About Alexion
Safe Harbor Statement
This news release contains forward-looking statements, including statements related to potential health and medical benefits of Soliris (eculizumab) for the treatment of patients with PNH. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, and a variety of other risks set forth from time to time in Alexion's filings with the
[ALXN-G]
References
(1) Abstract 271 entitled "Renal impairment is a risk factor for early mortality in patients with paroxysmal nocturnal hemoglobinuria (PNH)," presented by Dr.
(2) Abstract 254 entitled "Long term outcomes in patients with paroxysmal nocturnal hemoglobinuria (PNH) with sustained eculizumab treatment," presented by Dr.
(3) Abstract 841 entitled "Clinical impact of uncontrolled complement activity in Japanese non-transfused patients with paroxysmal nocturnal hemoglobinuria," presented by Dr.
(4) Abstract 833 entitled "Pediatric diagnosis of paroxysmal nocturnal hemoglobinuria in the International PNH Registry," presented by Dr. Alvaro Urbano-Ispizua at the 16th
(5) Socié G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996: 348:573-577.
(6) Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106 (12):3699-3709.
(7) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995; 333:1253-1258.
(8) Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
(9) Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal haemoglobinuria clones in patients with myelodysplastic syndromes. Br
(10) Maciejewski JP, Risitano AM, Sloand EM, et al. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br
Senior Director, Corporate Communications and Public Policy
or
Media:
or
Investors:
Source:
News Provided by Acquire Media