Soliris® and PNH

Soliris (eculizumab) is the first and only therapy specifically tested and approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, life-threatening blood disorder. Soliris has been approved for the treatment of patients with PNH in nearly 50 countries worldwide, including the United States (US), European Union (EU), and Japan.

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).

Chronic hemolysis is the main cause of serious health problems in people with PNH and can lead to blood clots, organ failure and shortened survival. In healthy individuals, red blood cells are equipped with a surface protein, known as a complement inhibitor, which protects them from naturally occurring levels of complement and prevents cell destruction. Patients with PNH lack the protective complement inhibitor protein, making red blood cells susceptible to low levels of complement activity, which leads to chronic hemolysis. As a result, the toxic contents of red blood cells are released into the bloodstream, causing many of the symptoms associated with PNH.

Historically, up to one third of patients with PNH treated with best supportive care did not survive beyond five years of diagnosis due to serious clinical outcomes such as thromboembolism (blockage of a blood vessel by a blood clot) and chronic kidney disease (CKD).

Treating Patients Who Have PNH with Soliris

Today, patients with PNH have the benefit of a therapy specifically tested and approved for the treatment of their condition. Soliris works by inhibiting terminal complement, which reduces hemolysis, improves symptoms and reduces many of the major health complications associated with PNH. Data from an independent long-term analysis in which patients were treated with Soliris for up to eight years show that Soliris dramatically alters the natural course of PNH and improves survival to the extent that it is equivalent to that of an age- and sex-matched normal control population.

To learn more about PNH, visit www.PNHSource.com. To learn more about treatment with Soliris, visit www.Soliris.net

Click here to view full prescribing information for Soliris.

IMPORTANT SAFETY INFORMATION
THE U.S. PRODUCT LABEL FOR SOLIRIS INCLUDES A BOXED WARNING

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

See full prescribing information for the complete boxed warning.

Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early (5.1).

  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.
  • Immunize patients with a meningococcal vaccine at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. (See Serious Meningococcal Infections (5.1) for additional guidance on the management of meningococcal infection.)
  • Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program (5.2). Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-soliris (1-888-765-4747).

In a randomized, placebo-controlled clinical study of Soliris in patients with PNH, the most frequently reported adverse reactions (≥ 10% overall and greater than placebo) were headache, nasopharyngitis (inflammation of the nasal passages and upper pharynx), back pain, and nausea.

In two prospective, single-arm studies of Soliris in patients with aHUS, the most frequently reported adverse reactions (≥ 15% combined per patient incidence) were hypertension (high blood pressure), upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia (abnormally low white blood cell count).