Kidney Patients with Alport Syndrome are Center of FDA Patient-Focused Meeting

Patients share with FDA experience of living with this rare form of kidney disease
Editor’s update Nov. 15, 2019:  The Voice of the Patient”  report from the Externally Led PFDD meeting on Alport syndrome has been posted by the U.S. Food and Drug Administration (FDA) on Nov. 1, 2019. The FDA will use this report to help inform their decisions on potential new treatments for Alport syndrome.
New York, NY—August 3, 2018—The National Kidney Foundation (NKF), along with The Alport Syndrome Foundation, are conducting an Externally-Led Patient-Focused Drug Development Meeting (PFDD) for a rare form of kidney disease, Alport syndrome (AS). Today’s meeting, which is only the second PFDD meeting of its kind focused on kidney disease, will bring together patients, care partners, and physicians along with representatives from the FDA to inform the Agency about the challenges of treating Alport syndrome and share the impact of living with this rare disease.  The meeting will be held today at the College Park Marriott Hotel and Conference Center in Hyattsville, MD. 
“We are honored to partner with the Alport Syndrome Foundation to organize today’s meeting. What is learned or even possibly discovered here can lay new foundations to benefit patients with AS,” said Kevin Longino, CEO, National Kidney Foundation and a kidney transplant patient.  “We believe this meeting will be a strong step forward to address the enormous unmet medical needs in the Alport syndrome community.”
Alport syndrome is a genetic disease that damages the tiny blood vessels in your kidneys. It can lead to kidney disease and kidney failure and can also cause hearing loss and problems within the eyes.  Alport syndrome causes damage to your kidneys by attacking the glomeruli, the tiny filtering units inside your kidneys. Alport syndrome is a rare inherited disease that only affects roughly 30,000-60,000 individuals in the United States, yet it accounts for 3% of children with chronic kidney disease and 0.2% of adults with end-stage renal disease (ESRD). It is caused by genetic mutations that affect the type IV collagen family of proteins. Type IV collagen is a major part of important tissue structures called basement membranes that are present in all tissues in the kidney, inner ear, and eye.
"Providing patients with an opportunity to speak directly to representatives of the FDA about what it’s really like living with this disease and outlining what AS patients need in terms of new therapies, will be critical to advancing much needed breakthroughs for those living with this rare form of kidney disease,” said Gina Parziale, CFRE, Executive Director, Alport Syndrome Foundation.
The Honorary Chair of the conference is Clifford Kashtan, MD, Professor, Department of Pediatrics and Fellowship Program Director and Faculty Member, Division of Pediatric Nephrology, University of Minnesota.
The conference is co-chaired by two leaders in Alport syndrome disease: Michelle Rheault, MD, Associate Professor, Department of Pediatrics, University of Minnesota and James Simon, MD, Assistant Professor, Cleveland Clinic Lerner College of Medicine, Cleveland Clinic.
Conference speakers include: Alport syndrome patients, care partners and patient representatives; Ellis Unger, MD, Director of the Office of Drug Evaluation-I, FDA; Aliza Thompson, MD, MS, Clinical Team Leader in the Division of Cardiovascular and Renal Products, Center for Drug Evaluation and Research (CDER), FDA; Sharon Lagas, President, Alport Syndrome Foundation; Gina Parziale, CFRE, Executive Director, Alport Syndrome Foundation; David Feldman, PhD, Medical Project Director, National Kidney Foundation.
To participate in today’s conference via webinar register here.
Sponsorship for the Externally Led Patient-Focused Drug Development Meeting on Alport Syndrome was provided by Reata, Retrophin, Boerhinger Ingelheim-Lilly, and Regulus.
Kidney Disease Facts
30 million American adults are estimated to have chronic kidney disease—and most aren’t aware of it.  1 in 3 American adults are at risk for chronic kidney disease.  Risk factors for kidney disease include diabetes, high blood pressure, heart disease, obesity and family history.  People of African American, Hispanic, Native American, Asian or Pacific Islander descent are at increased risk for developing the disease.  African Americans are 3 times more likely than Whites, and Hispanics are nearly 1.5 times more likely than non-Hispanics to develop end stage renal disease (kidney failure).
The National Kidney Foundation (NKF) is the largest, most comprehensive and longstanding organization dedicated to the awareness, prevention and treatment of kidney disease. For more information about NKF visit
The Alport Syndrome Foundation is a non-profit organization founded in 2007 by a group of families affected by the disease and guided by a Medical Advisory Committee of renowned Nephrologists. The Foundation was built on the past efforts of the late Dr. Curtis Atkin (who himself had Alport syndrome) of the University of Utah and the Hereditary Nephritis Foundation.  ASF is the leading independent non-profit organization in the United States serving and giving a voice to the Alport syndrome community.