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FDA: orphan drug designation to Soliris for prevention of delayed graft function in renal transplant patients


The FDA ( Food and Drug Administration ) has granted an orphan drug designation ( ODD ) to Soliris ( Eculizumab ), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function ( DGF ) in renal transplant patients.
DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation. In patients undergoing a kidney transplant, delayed graft function leads to the patient requiring dialysis in order to survive.

Soliris is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria ( PNH ) and atypical hemolytic uremic syndrome ( aHUS ), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
Soliris is not approved in any country to prevent or treat DGF following kidney or other solid organ transplantation.

Delayed graft function is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation.
In the case of DGF in the setting of kidney transplantation, the patient requires dialysis after the transplantation procedure. Most often, delayed graft function results from organ injury caused by reduction and/or restoration of blood flow, and the associated inflammation, including complement activation.
Delayed graft function 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.

In addition, as kidney donors are in short supply, reducing the risk of delayed graft function may allow more donor kidneys to be transplanted.
At present, 15-20% of donor kidneys are reportedly never used and thus discarded each year in the U.S. and Europe due to the risk of poor outcomes associated with delayed graft function, denying many patients the benefit of solid organ transplantation.

The U.S. product label for Soliris includes a 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.
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 two weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection.
Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis, back pain and nausea.
Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.

In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were hypertension, upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia. ( Xagena )

Source: Alexion, 2014

XagenaMedicine_2014



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