FDA: 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.