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.
Biochemists, microbiologists, drug discovery experts and infectious disease doctors have teamed up in a new study that shows antibiotics are not always necessary to cure sepsis in mice. Instead of killing causative bacteria with antibiotics, researchers treated infected mice with molecules that block toxin formation in bacteria.READ MORE
Previous work by the International Multiple Sclerosis Genetics Consortium (IMSGC) has identified 233 genetic risk variants. However, these only account for about 20% of overall disease risk, with the remaining genetic culprits proving elusive. A new study has tracked down four of these hard-to-find genes.READ MORE
The sweet flavors’ appeal to teens is a major concern for Food and Drug Administration officials, who recently declared teen vaping an epidemic. New research shows flavorings are transforming more than marketing. The chemical additives react to e-liquid, or e-juice, creating new compounds that could trigger irritation and inflammation when inhaled.READ MORE