Editor’s Note: The FDA approval of the Liposorber LA-15 System is an activity that is funded under FDA’s user fee program in the Center for Devices and Radiological Health (CDRH). Some FDA activities that do not rely on annual appropriations are continuing during the government shutdown.
FDA NEWS RELEASE
For Immediate Release: Oct. 10, 2013
Media Inquiries: Steven Immergut, 301-796-7507, email@example.com
Consumer Inquiries: 888-INFO-FDA
FDA approves new pediatric use for Liposorber Apheresis System
The U.S. Food and Drug Administration today approved Liposorber LA-15 System to treat pediatric patients with primary focal segmental glomerulosclerosis (FSGS) either before transplant, or after renal (kidney) transplantation in which there is recurrence of FSGS.
FSGS is a chronic disease in which scar tissue develops on the parts of the kidneys that filter waste out of the blood and in other essential parts of the kidney. FSGS causes excessive loss of protein from the blood into the urine, which may lead to nephrotic syndrome (edema, excess protein in the urine, low protein in the blood, and high cholesterol) and kidney failure. A majority of children with primary FSGS will progress to end stage renal disease and will require either kidney dialysis or a kidney transplant. About one quarter to one half of FSGS patients that receive a kidney transplant will have a recurrence of FSGS in their transplanted kidney.
“Primary FSGS is a rare disease,” said Christy Foreman, director of the Office of Device Evaluation at the FDA’s Center for Devices and Radiological Health. “This approval provides a treatment option for children with primary FSGS when standard treatment options, including diet modifications and drug therapies, are unsuccessful or not well tolerated.”
The Liposorber LA-15 System, a blood processing system that is used outside the body, includes disposable components and a control/monitor unit. The device works by removing certain lipoproteins from the patient’s blood. The patient’s blood is first passed through a plasma filter where the blood cells are separated from plasma (the liquid component of the blood). The plasma is then further passed through two adsorption columns, which are packed with a gel designed to capture the lipoproteins in the blood. The blood cells and the treated plasma are then returned to the patient via the blood return line.
The Liposorber LA-15 System, marketed by New York-based Kaneka America Corporation, was first approved in the United States in 1996. The FDA approved it for lowering low density lipoprotein cholesterol (often called the “bad” cholesterol) in certain patients with familial hypercholesterolemia (FH) who develop very high cholesterol levels, increasing the risk for complications from the disease, including cardiovascular disease. FH patients treated with the device are those for whom dietary and drug therapies have not been effective, or for whom drug therapy cannot be tolerated.
Data in support of approval included two scientific studies – one published in a peer-reviewed scientific journal and one unpublished report. One study of 11 children with FSGS at high risk for progression to end stage renal disease showed normal or near normal renal function in most patients who achieved remission of disease after treatment with the Liposorber LA-15 System. The study authors reported only one adverse event. The other study showed that patients with FSGS who received therapy with the Liposorber LA-15 System after a kidney transplant (including some who received one treatment immediately before transplant) excreted less protein in their urine than FSGS patients who underwent a kidney transplant but received no therapy with the Liposorber LA-15 System. These study results, along with non-clinical data submitted by the company, support a determination that the Liposorber LA-15 System has probable benefit for pediatric patients for whom standard FSGS treatments are too risky, ineffective, or cannot be tolerated, and, that the device does not expose patients to unreasonable risks.
The Liposorber LA-15 System indication for pediatric patients is designated as a Humanitarian Use Device (HUD) by the FDA’s Office of Orphan Products Development. Medical devices are eligible for HUD designation if they are intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in fewer than 4,000 individuals in the United States per year. Devices that receive HUD designations may be eligible for marketing approval under the Humanitarian Device Exemption (HDE) marketing pathway. The Liposorber LA-15 System indication for pediatric patients is approved for pediatric use through the HDE marketing pathway.
The FDA approval of an HDE authorizes an applicant to market the device subject to certain profit and use restrictions. After the passing of the Pediatric Medical Device Safety and Improvement Act of 2007, original HDE applications for devices that are indicated for use in pediatric patients or in a pediatric subpopulation and are approved on or after Sept. 27, 2007 and assigned an annual distribution number are permitted to be marketed for profit.
For more information:
FDA: Designating Humanitarian Use Devices
NIH: National Institute for Diabetes and Digestive and Kidney Diseases: Glomerular Disease Primer: Selected Glomerular Diseases:
NIHL Rare Diseases Network: Focal and Segmental Glomulerosclerosis
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.