Kidney Patients with C3G are Center of FDA Patient-Focused Meeting

Patients share with FDA experience of living with this rare form of kidney disease
New York, NY—August 3, 2017—The National Kidney Foundation (NKF), along with the Food and Drug Administration (FDA), is conducting an Externally-Led Patient-Focused Drug Development Meeting (PFDD) for a rare form of kidney disease, complement 3 glomerulopathy or C3G. This is the first-ever PFDD meeting focused on kidney disease. The upcoming meeting, to be held August 4th from 8:30 a.m. to 1:10 p.m., at the Tommy Douglas Conference Center in Silver Spring, MD, will bring together patients, caregivers, physicians who are experts in C3G and representatives from the FDA to learn about the impact of living with this rare disease.

“The goal of NKF’s patient-focused meeting on C3G is to give patients the opportunity to explain to the FDA how this disease affects them personally so that the agency can understand the disease’s unique burdens and why developing effective medicines is urgently needed,” said Joseph Vassalotti, MD, Chief Medical Officer, NKF. “There are currently limited drug therapies available and no effective, targeted treatment options for the roughly 4,000 Americans living with C3G disease. Working together with the FDA, and patients affected by C3G, our goal is to advance treatment innovations.”

C3G is a rare disease typically striking patients in the prime of life; and their prognosis is poor. The average age for diagnosis is 21-26 years, and approximately 30-50% of C3G patients will progress to end-stage kidney disease (the stage where kidneys fail) within 10 years of diagnosis. Once kidneys fail, dialysis or transplant is needed just to stay alive. Unfortunately for patients with C3G, kidney transplantation is not highly successful as the disease recurs in more than 50% of patients post-transplant, requiring a return to dialysis or a second transplant that is likely to have the same poor outlook as the first. Dialysis patients on average live approximately 15 years less than a person with a successful kidney transplant. C3G is characterized by variable amounts of proteinuria (protein in urine), hematuria (blood in urine), hypertension and decreased kidney function.

“I was diagnosed with C3G when I was just seven years old, and have spent the last 22 years on dialysis,” said 31-year-old Jenna Smith of Seattle Washington, a panelist at the meeting. “While I strive to be like most people my age, I’m just not, the daily responsibilities of existing with this disease do not allow me to be. I’m hoping that by participating in the C3G meeting, representatives from the FDA and pharmaceutical companies will understand why I matter, and why people like me are worth their investment of time and research.”

In conducting research for the conference NKF polled patients anonymously and asked what advice they would give to a drug company to help develop a new medicine for C3G; and what advice they would give to the FDA to help them decide whether to approve it. Some responses included:

  • “…..drug companies need to realize that they are developing drugs for a very small population and that these patients are already incurring a great deal of medical debt.  We cannot afford millions of dollars to stay alive, or to keep our children alive.”
  • “Patients suffer a great deal with side effects from ineffective drug alternatives and their health deteriorates as a result of ineffective treatment.”
  • “….many patients are looking at a lifetime on dialysis, which is not a pleasant way of life, nor can most people sustain it for more than a few years.  A new drug certainly comes with risks—but it also comes with a great deal of hope and possibility.”
  • “…..if a drug can be shown to be effective for even a portion of C3G patients and can be deemed safe, then put it in the hands of the people who might benefit.  They don’t have any good options, so why not give them a chance?”
The nearly five-hour conference will be co-chaired by two leading nephrologists specializing in C3G: Carla Nester, MD, Assistant Professor of Medicine and Pediatrics, University of Iowa, Carver College of Medicine, Iowa City, IA and Andrew S. Bomback, MD, MPH, Assistant Professor of Medicine, Columbia University Medical Center, New York, NY. View the agenda or participate in the conference via webinar.

FDA speakers include Jonathan Goldsmith, MD, Associate Director of Rare Diseases Program, Center for Drug Evaluation and Research (CDER), Office of New Drugs; Aliza Thompson, MD, Clinical Team Leader, Division of Cardiovascular and Renal Products, CDER, and Normal L. Stockbridge, MD, PhD; Division Director, Cardiovascular and Renal Products.  

Lead sponsorship for NKF’s Patient-Focused Drug Development Meeting was provided by Achillion Pharmaceuticals. Additional support was provided by Novartis Pharmaceuticals. 

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