Sickle Cell Biologic Granted Orphan Drug Designation
The FDA has granted orphan drug designation to Prolong Pharmaceuticals' sickle cell disease treatment.
The FDA has granted orphan drug designation to Prolong Pharmaceuticals’ sickle cell disease (SCD) treatment.
Sanguinate is an experimental biologic with antivasoconstrictive properties that facilitate the transfer of oxygen to oxygen-deprived cells and tissues and down-regulate blood vessel inflammation. It is the only biological product currently under clinical development for SCD’s debilitating, acute co-morbidities, which include sickle cell crisis, acute chest syndrome, leg ulcers, and pediatric and adult stroke.
“Our orphan drug designation has been issued based in part on Sanguinate’s unprecedented ability to un-sickle sickle cells,” said Prolong Pharmaceuticals president Glenn Kazo in a press release.
With its orphan drug designation, Sanguinate will be awarded a period of marketing exclusivity upon FDA approval, among other benefits and incentives.
Multiple clinical trials on Sanguinate are underway in an attempt to establish its safety and efficacy in SCD and other diseases caused by ischemia, hypoxia and/or hemolysis. Currently, the drug is undergoing a phase 2 study in reducing or preventing delayed cerebral ischemia following subarachnoid hemorrhage, with more phase 2 trials planned for vaso-occlusive crisis and leg ulcers secondary to SCD, as well as for preventing delayed graft function following kidney transplantation.