For Immediate Release
March 6, 2015
The U.S. Food and Drug Administration today authorized use of Lixelle Beta 2-microglobulin Apheresis Column, the first device to treat dialysis-related amyloidosis (DRA).
Dialysis-related amyloidosis is a chronic, progressive condition caused by the buildup in the body of a protein called beta 2-microglobulin. Dialysis-related amyloidosis is a complication of kidney failure. As beta 2-microglobulin builds up in the blood, deposits of the protein can form in the bones, joints and tendons causing painful and stiff joints, bone cysts that can lead to bone fractures, and torn tendons and ligaments. Beta 2-microglobulin deposits can also affect the digestive tract and organs, such as the heart and lungs.
Dialysis-related amyloidosis most often occurs in patients with kidney failure, especially adults older than 60, who have been on hemodialysis for more than five years.
The Lixelle Column works by removing beta 2-microglobin from the blood. It contains porous cellulose beads specifically designed to bind to beta 2-microglobulin as the patient’s blood passes over the beads. The device is used in conjunction with hemodialysis, a treatment where blood circulates outside the body through a special filter that removes waste products and extra fluid. The clean blood is returned to the body. When the Lixelle Column is used, the blood passes through the Lixelle Column before it enters the dialysis filter.
The device may help patients who have developed symptoms related to DRA and may be especially useful for those patients who may not have access to extended dialysis therapies or who may not be eligible for a kidney transplant.
“While DRA affects only a small population of patients on dialysis, there are not many treatment options for these patients and some options may not be available to patients in all areas,” said William Maisel, M.D., M.P.H., deputy director for science, chief scientist and acting director of the Office of Device Evaluation in FDA’s Center for Devices and Radiological Health. “The Lixelle Beta 2-microglobulin Apheresis Column may provide this patient population with an option for relieving some of the debilitating symptoms of DRA.”
The FDA reviewed the Lixelle Column through the Humanitarian Device Exemption (HDE) pathway after granting it a Humanitarian Use Device (HUD) designation. An HDE is an application that is similar to a premarket approval application (PMA), but it is exempt from the effectiveness requirements that apply to PMAs. Devices are eligible for HUD designation if they are designed to treat or diagnose a disease or condition that affects or is manifested in fewer than 4,000 individuals in the U.S. per year. In order to receive HDE approval for a HUD, a company must demonstrate safety and probable benefit of the device (i.e., that the device will not expose patients to an unreasonable or significant risk of illness or injury, and that the probable benefit of the device outweighs the risk of injury or illness from its use), and that there are no legally-marketed comparable devices, other than a device approved under the HDE or investigational device exemption (IDE), available to treat or diagnose the disease or condition.
Data supporting the safety and probable benefit of the Lixelle Column include published clinical studies describing treatment of approximately 100 patients from Japan with DRA, and postmarket safety data from approximately 200 patients in Japan where the device has been approved for use. The studies generally showed improvement in symptoms associated with DRA with use of the device.
The most common adverse events associated with the device’s use are temporary hypotension (low blood pressure) and a decrease in red blood cell count (hematocrit). These are common adverse events for patients undergoing dialysis or any extracorporeal therapy (treatment that takes place outside of the body). As a condition of the HDE approval, the company must conduct a postmarket study to gain more data on the benefits, risks, and adverse events in the U.S. population.
The Lixelle Column is manufactured by Kaneka Corporation in Osaka, Japan and distributed in the U.S. by its subsidiary, Kaneka Pharma America.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.