Alport Syndrome and Its Challenges Focus of Early August Meeting with FDA

The National Kidney Foundation (NKF) recently held a patient-focused drug development meeting concerning Alport syndrome (AS) with the U.S. Food and Drug Administration (FDA). This meeting was conducted in conjunction with The Alport Syndrome Foundation.

The meeting, which was took place Aug. 3 in Hyattsville, Maryland, was the second externally-led, patient-focused drug development (PFDD) meeting about kidney disease.

It brought patients, caregivers, family members and healthcare professionals together to share with the FDA and with pharmaceutical industry representatives their viewpoints regarding the many challenges that underlie treating Alport syndrome, and the difficulties in living with the disease and its impact on everyday life.

“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,” is critical to  “advancing much needed breakthroughs for those living with this rare form of kidney disease,” Gina Parziale, CFRE, executive director of the Alport Syndrome Foundation, said in a press release.

Kidney damage in Alport syndrome is the result of damage to the glomeruli – a cluster of small blood vessels inside the kidneys — and the disease can lead to kidney failure.

Dr. Clifford Kashtan, a professor in the department of pediatrics at the University of Minnesota, served as the honorary chair of the conference. Two leading Alport researchers — Dr. Michelle Rheault, an associate [rofessor at the University of Minnesota and Dr. James Simon, an assistant professor at the Cleveland Clinic Lerner College of Medicine — chaired the meeting.

Information gathered at the meeting, both from comments and from surveys requested from patients and other participants, will be used by the FDA and by pharmaceutical companies in decisions regarding possible treatments and in research that might lead to new treatments, the foundation states on a webpage.

Patients and their families are also invited to take part in the next Alport Connect meeting, set for Sept. 29–30 at University of Texas Southwestern. Registration deadline is Aug. 29, and travel stipends may be available for qualifying families. More information is available on the foundation’s upcoming events page, or by clicking here.