An investigational drug being developed for the treatment of autosomal dominant polycystic kidney disease (ADPKD) has received orphan drug designation by the U.S. Food and Drug Administration (FDA). Lixivaptan is a potent, selective vasopressin V2 receptor antagonist which may delay the progression of ADPKD.
Lixivaptan was previously administered to 1,673 patients across 36 clinical studies as part of a prior clinical development program for the treatment of hyponatremia. Palladio Biosciences, Inc., who owns the drug, expects to leverage the data generated in the hyponatremia clinical trial to repurpose lixivaptan and advance its development for the treatment of ADPKD.
“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.”
Orphan drug designation was created by the FDA in 1983 to incentivize pharmaceutical companies to develop treatments for rare diseases.
According to the FDA, the Orphan Drug Designation program provides orphan status to drugs intended for treatment of a disease or disorder diagnosed in fewer than 200,000 people. Although 600,000 people are estimated to have ADPKD in the U.S., it is a highly under diagnosed disease. The orphan figure is based on actual diagnosed cases of the disease.
Pharmaceutical companies who have been granted orphan designation for investigational drugs can receive various benefits including accelerated registration and certain tax credit and marketing incentives. So if lixivaptan were to be approved for its use on ADPKD, Palladio would have reduced fees and tax breaks, decreasing the price of bringing the drug to the market.
Additionally, this recognition of ADPKD as an orphan (rare) disease could potentially mean more options for PKD patients as it may encourage more pharmaceutical companies to develop drugs to treat ADPKD. An orphan disease status may also result in the potential of faster and less expensive drug development. All treatments, however, will still be required to meet FDA standards.