Investigational Treatment for ADPKD Earns FDA’s Orphan Drug Designation

Approximately 540,000 individuals in the United States have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD).

The disease causes persistent and rapidly accumulating cysts to grow in the patient’s kidneys. They are usually benign.

Cysts may also grow in the pancreas, liver or other regions of the body. For a large majority of patients, the number of cysts increases to a point that organs fail, and either a transplant or dialysis may be necessary.

About Orphan Drug Designation (ODD)

Last week, according to a press release published in Biospace, Dr. Gavin Xia, the Co-founder and CEO of Alebund Pharmaceuticals, Shanghai, announced that the FDA granted ODD to AP303 for the treatment of ADPKD.

ODD is assigned to drugs that show potential in treating a rare disease affecting less than 200,000 individuals in the US.

The drug companies receiving the designation are incentivized by receiving tax credits to assist with the cost of clinical research, marketing exclusivity, exemption from certain fees, and other benefits that would encourage the development of orphan drugs.


ADPKD is most often caused by mutations in the PKD1 gene. It is the most common genetic disorder caused by a single mutation and one of the foremost causes of kidney failure worldwide. Monogenic disorders result from inheriting a single gene mutation from either parent.

The term “autosomal dominant” translates to one parent having the genetic variation that causes the disease and each child having a 50% chance of inheriting the disorder.

The drug has demonstrated improvements in renal survival in ADPKD mouse models. AP303 has now completed its first-in-human study of healthy subjects in Australia. The drug is now ready for an ADPKD Phase II study.

ADPKD is a major cause of end-stage kidney disease. It affects somewhere between 1 in 400 to 1 in 1,000 live births. Many of these children will, at some point, require renal replacement therapy.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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