– Orphan drug designation an important milestone for lixivaptan development program –
Newtown, PA: October 4, 2017. Palladio Biosciences, Inc. (Palladio), a privately held biopharmaceutical company founded to develop medicines that make a meaningful impact on the lives of patients with orphan diseases of the kidney, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to lixivaptan for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).
There are currently no drug treatments approved for ADPKD in the United States.
The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 people but are not expected to recover the costs of drug development and marketing. Orphan drug designation provides eligibility for certain benefits, including seven years of market exclusivity following receipt of regulatory approval, tax credits for qualified clinical trials, and exemption from FDA application fees.
“ADPKD is a serious progressive, inherited disease that typically affects multiple generations of entire families. Lixivaptan has the potential to slow the progression of ADPKD and possibly delay the need for dialysis or a kidney transplant,” said Lorenzo Pellegrini, Ph.D., Founder and Chief Executive Officer of Palladio. “The granting of orphan drug designation is an important milestone in the lixivaptan development program.”
Lixivaptan is a potent, selective vasopressin V2 receptor antagonist. This mechanism of action has clinical proof of concept to delay the progression of the autosomal dominant form of PKD. Lixivaptan was previously administered to 1,673 subjects across 36 clinical studies as part of a prior clinical development program for the treatment of hyponatremia. Palladio expects to leverage lixivaptan’s large body of data generated in the hyponatremia clinical program to repurpose lixivaptan and advance its development for the treatment of ADPKD.
About Polycystic Kidney Disease (PKD) – Key Facts and Figures:
PKD is an inherited genetic disease that affects thousands of people in the United States and millions globally. ADPKD is the most common type of PKD. A person with ADPKD has a 50 percent chance of passing the disease on to each of his or her children. The disease is characterized by uncontrolled growth of fluid-filled cysts in the kidney, which can each grow to be as large as a football. Symptoms often include kidney infections and chronic pain. The continued enlargement of cysts and replacement of normal kidney tissue causes irreversible loss of renal function. In the United States, approximately 2,500 new people with PKD require dialysis or a kidney transplant every year, making PKD the 4th leading cause of kidney failure. There is no cure for PKD.
About Palladio Biosciences, Inc.:
Palladio Biosciences is a privately-owned, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney and is located in Newtown, PA. For more information, please visit www.palladiobio.com.
Please note that lixivaptan is for investigational use only.
Palladio Biosciences, Inc.
+1 908 294 8728