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First Device to Treat Patients With Dialysis-Related Amyloidosis Gets FDA Nod

Apheresis column removes beta 2-microglobulin proteins

The FDA has authorized the use of the Lixelle beta 2-microglobulin apheresis column (Kaneka Pharma America), the first device to treat dialysis-related amyloidosis (DRA).

DRA is a chronic, progressive condition caused by the buildup in the body of beta 2-microglobulin proteins. Dialysis-related amyloidosis is a complication of kidney failure. As beta 2-microglobulins accumulate 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 other organs, such as the heart and lungs.

DRA occurs most often in patients with kidney failure, especially adults more than 60 years of age who have been on hemodialysis for more than 5 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 in which blood circulates outside the body through a special filter that removes waste products and extra fluid. The clean blood is then returned to the body. When the Lixelle column is used, the blood passes through the device before it enters the dialysis filter.

The Lixelle column 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, according to the manufacturer.

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 an “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. To receive HDE approval for an HUD, a company must demonstrate the safety and probable benefit of the device, and it must show that no legally marketed comparable devices, other than a device approved under the HDE or investigational device exemption (IDE), are 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 Japanese patients with DRA, and post-marketing safety data from approximately 200 Japanese patients in whom the device had been approved for use. The studies generally showed an improvement in DRA symptoms with use of the device.

The most common adverse events associated with the use of the Lixelle column included temporary hypotension and a decrease in the hematocrit count. These are common adverse events for patients undergoing dialysis or any extracorporeal therapy. As a condition of the HDE approval, the manufacturer must conduct a post-marketing study to obtain more information on the benefits, risks, and adverse events associated with the Lixelle column in U.S. patients.

Source: FDA; March 6, 2015.

 

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