Alexion Pharmaceuticals

Soliris^® and aHUS

Soliris is the only therapy approved for the treatment of atypical hemolytic uremic syndrome (aHUS), an ultra-rare, life-threatening, genetic disease that can progressively damage vital organs, leading to stroke, heart attack, kidney failure and death. Soliris is indicated for the treatment of patients with aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA). The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga toxin E.Coli-related hemolytic uremic syndrome (STEC-HUS).

About aHUS

aHUS is caused by a permanent genetic mutation that leads to uncontrolled, and excessive activation of complement, leading to the formation of blood clots in small blood vessels throughout the body.^4,12,13 These blood clots are known as thrombotic microangiopathy, or TMA, and they can lead to life-threatening damage to vital organs, including the kidneys, heart and brain.^4,5 aHUS affects both adults and children, with about 60 percent of patients diagnosed under the age of 18.¹⁴

Patients with aHUS face a lifelong risk of sudden, progressive, life-threatening complications due to TMA.¹⁵ In the past, using prior disease management strategies, more than one-half of patients with aHUS died, required kidney dialysis, or had permanent renal damage within one year of diagnosis.¹⁵

Treating aHUS with Soliris

Prior to the approval of Soliris for the treatment of aHUS, doctors relied on disease management strategies that do not specifically target uncontrolled complement activation, the underlying cause of TMA in patients with aHUS. Soliris represents a major step forward in the care of patients with aHUS. In clinical studies,100% of patients had a reduction in terminal complement activity with sustained treatment with Soliris.^16,17 By considerably reducing TMA, Soliris improved kidney function, reduced the need for interventions such as dialysis, and substantially improved quality of life in the studied patients.^16,17

More Information

To learn more about aHUS, visit www.aHUSsource.com. To learn more about treatment with Soliris, visit www.Soliris.net.

IMPORTANT SAFETY INFORMATION

The U.S. product label for Soliris includes a boxed warning:

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

See full prescribing information for 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).

The most frequently reported adverse reactions in the PNH randomized trial (≥ 10% overall and greater than placebo) are: headache, nasopharyngitis, back pain, and nausea.

The most frequently reported adverse reactions in aHUS single arm prospective trials (≥ 15% combined per patient incidence) are: hypertension, upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia.

Please see full Prescribing Information and Important Safety Information for Soliris^® (eculizumab).