FDA Grants Orphan Drug Status for Quark's QPI-1002 for the Prophylaxis of DGF in Kidney Transplant Patients
News Feb 17, 2010
Quark Pharmaceuticals, Inc. has announced 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.
The cholesterol-lowering drugs called statins have demonstrated substantial benefits in reducing the risk of heart attacks and strokes caused by blood clots (ischemic strokes) in at-risk patients. Since statins are associated with a low risk of side effects, the benefits of taking them outweigh the risks, according to a scientific statement from the American Heart Association that reviewed multiple studies evaluating the safety and potential side effects of these drugs.READ MORE
18th International Conference on Pharmaceutics & Novel Drug Delivery Systems
May 27 - May 28, 2019