Quark Pharmaceuticals, Inc., a company involved in the discovery and development of RNAi-based therapeutics, announced today that the US FDA has granted orphan drug designation for QPI-1002 (also referred to as “I5NP”), a synthetic siRNA targeting p53 mRNA for the prophylaxis of delayed graft function (DGF) in kidney transplant patients. Quark has completed enrollment and dosing in the dose-escalation safety portion (Part A) of a Phase 1/2 study and has received a positive recommendation from an independent Data Safety Monitoring Board (DSMB) to progress the drug to the second portion (Part B) of the study to evaluate the safety and potential clinical activity of QPI-1002.
“DGF is currently an unmet medical need, and we anticipate a great deal of interest in QPI-1002 from the renal transplant community. We are confident that obtaining orphan status will facilitate the rapid development of this innovative compound and hope that ultimately, we can reduce the wait time for kidney transplants with fewer failed grafts,” Daniel Zurr, Ph.D., President and Chief Executive Officer of Quark Pharmaceuticals commented.
US orphan drug designation is granted to products intended to treat a rare disease or condition affecting less than 200,000 Americans. The Orphan Drug Act allows for possible R&D incentives and qualifies the sponsor for tax credits and a period of market exclusivity following approval.