Alport Syndrome Patients Met With the FDA About Drug Development

According to a story from PR Newswire, patients with Alport syndrome, supported by the National Kidney Foundation and the Alport Syndrome Foundation, were involved in a meeting with representatives from the FDA, physicians and care partners. This is the second kidney disease focused meeting that will primarily focus on drug development. Patients shared about potential challenges of treating Alport syndrome as well as what it is like to live with this rare disease.

About Alport Syndrome

Alport syndrome is a genetic disorder which is characterized by end stage kidney disease, hearing loss, and glomerulonephritis, a type of kidney disease in which the glomeruli or small blood vessels in the kidneys become inflamed. Alport syndrome is a heritable disease, and is caused by mutations of the COL4A3, COL4A4, and COL4A5 genes. The symptoms of the disorder include blood and protein in the urine, leiomyomatosis (coughing, vomiting, bronchitis, stomach pain, dysphagia), various eye conditions, and, rarely, aortic dissection. Since kidney failure inevitably develops eventually, treatment is similar to that of chronic kidney disease, and patients will require dialysis or a kidney transplant. ACE inhibitors appear to slow the decline of kidney function. To learn more about Alport syndrome, click here.

The Patient’s Role in Drug Development

Kevin Longino, the CEO of the National Kidney Foundation, believes that the meeting was a “strong step forward” in moving towards addressing the unmet needs of the Alport syndrome patient community. Unfortunately, it is unusual for patients to get an opportunity to directly speak with FDA representatives, and feedback from patients will hopefully play a central role in developing new treatments.

New Therapies Needed

The meeting also involved many Alport syndrome specialists from across the country, who should also have valuable experience in treating the disorder. Treatment options for Alport syndrome are limited, and since it is the cause of only 0.2 percent of adult cases of end stage kidney disease, it often gets overlooked. Patients with this disorder need more specialized treatment that will prevent them from having to deal with the consequences of kidney failure.

Hopefully, this meeting has served as the first step in getting new therapies for Alport syndrome to the drawing board.

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