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Eculizumab in Other Diseases


Antibody-Mediated Rejection (AMR)

Acute antibody-mediated rejection (AMR) is a severe and potentially life-threatening condition that can lead to severe kidney allograft damage, resulting in rapid loss of function and possible loss of the transplanted kidney.1 Research suggests that uncontrolled complement activation, triggered by the binding of donor-specific antibodies (DSAs) to target proteins in the donor kidney, may be the primary cause of acute AMR in kidney transplant recipients.1,2

Sensitized patients—those who have high levels of DSAs—are at high risk for developing AMR.1,2 Approximately one-third of patients on the kidney transplant waiting list are sensitized to their potential donors3 and, historically, approximately 30% of this highly sensitized population has developed AMR.4 Due to the risk of AMR, sensitized patients may have difficulty finding a viable donor organ, and therefore may never become eligible for transplantation or may face significant delays in finding a suitable transplant. Instead, these patients remain on dialysis, which carries a very high mortality risk. In fact, approximately 65% of patients die within 5 years of commencing dialysis.5

Currently, there are no approved therapies for the prevention or treatment of AMR. Alexion is investigating eculizumab in patients receiving kidney transplants who are at elevated risk of AMR. Visit to learn more.

Delayed Graft Function (DGF)

Delayed graft function (DGF) is an early and serious complication of organ transplantation in which a transplanted kidney, known as a graft, does not function normally immediately following transplantation.6,7 Patients experiencing DGF after a kidney transplant require dialysis in order to survive.8-10

DGF affects approximately 25 to 50 percent of deceased-donor kidney transplant cases.6,7 Most often, DGF results from organ injury caused by severe inflammation and complement activation associated with the normal processes for removal, storage, and transplantation of the donor organ.11-15 DGF has a substantial negative impact on graft function both in the short and long term, which can result in premature graft loss, prolonged hospitalization or patient death.16,17

There remains an urgent need for innovative therapies to prevent DGF, which currently has no approved treatment options.18 Because donor organs are in short supply, reducing the risk of DGF for organs that are at higher risk of developing this devastating complication may allow more donor organs to be transplanted.19,20

Alexion has a clinical trial under way called the PROTECT (PReventiOn of delayed grafT function using ECulizumab Therapy) Study to evaluate the efficacy and safety of eculizumab in preventing DGF after kidney transplant in adult patients at increased risk of DGF. Visit (Identifier NCT02145182) to learn more about PROTECT.


Neuromyelitis Optica Spectrum Disorder (NMOSD)

Neuromyelitis optica spectrum disorder (NMOSD) is a life-threatening, ultra-rare autoimmune neurological disorder that damages the spinal cord (known as transverse myelitis) and the optic nerve (optic neuritis).21-23 NMOSD is caused by binding of the NMO antibody to receptors in the central nervous system (CNS). This triggers an inflammatory process that activates the complement system, leading to the destruction of cells in the CNS.24

Patients with NMOSD may experience unpredictable attacks of transverse myelitis and optic neuritis that can be severe, leading to permanent disability and in some cases death.25-29 There are no approved therapies for NMOSD, and unmet needs are high. More than 80% of patients experience relapses; 41% are legally blind in one eye; 22% require the use of a cane; and 8% need a wheelchair and are unable to walk.25,30 Immunosuppressant therapies may help reduce the occurrence of attacks but none have direct effects on the complement system, which is a central driver of the clinical attacks in patients with NMOSD.30, 31

Alexion is currently enrolling patients in the PREVENT (Prevention of Relapses and Evaluation of Eculizumab in NMO Treatment) study to evaluate the safety and efficacy of eculizumab as a potential treatment for patients with relapsing NMOSD. To learn more about PREVENT, visit (Identifier NCT01892345) visit, or call 1-855-687-1988.

Myasthenia Gravis (MG)

Myasthenia gravis (MG) is a rare, debilitating, complement-mediated neurological disorder in which the body’s immune system initiates an attack on the neuromuscular junction (NMJ). This attack causes tissue damage and impaired neuromuscular transmission, which can manifest in patients as debilitating weakness and/or fatigue.32,33 Many patients with MG initially experience weakness in their ocular (eye) muscles, and the disease typically progresses to the more severe and generalized form to include head, spinal, limb and respiratory muscles.34 Symptoms can include drooping eyelid, blurred vision, slurred speech, difficulty chewing or swallowing, weakness in the arms and legs, and difficulty breathing, which could lead to a life-threatening myasthenic crisis.34,35

There is no known cure for MG, and current management approaches do not completely address the localized, complement-driven attack on the neuromuscular junction.33,36 Today, approximately 10% to 15% of MG patients are considered refractory—meaning that despite current treatment, they experience intolerable side effects or continue to experience debilitating muscle weakness that severely impairs their ability to engage in simple daily activities such as speaking, swallowing, chewing and even breathing normally.36,37 

Alexion is conducting the REGAIN study (Eculizumab for REfractory GenerAlIzed MyastheNia Gravis) to evaluate the safety and efficacy of eculizumab as a potential treatment for patients with refractory generalized MG. Enrollment in this study is complete. To learn more about REGAIN, visit (Identifier NCT01997229), visit or call 1-855-687-1988.


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  2. Collins AB, Schneeberger EE, Pascual MA, et al. Complement activation in acute humoral renal allograft rejection: diagnostic significance of C4d deposits in peritubular capillaries. J Am Soc Nephrol. 1999;10(10):2208-14.
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  19. US Organ Procurement and Transplantation Network/Scientific Registry of Transplant Recipients. OPTN/SRTR Annual Report, 2009. Chapter II: Organ donation and utilization in the United States, 1999-2008.
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