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FDA grants orphan drug designation to Sanguinate for sickle cell disease
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The FDA granted orphan drug designation to PEGylated carboxyhemoglobin bovine for the treatment of sickle cell disease, according to a press release from the drug’s manufacturer.
PEGylated carboxyhemoglobin bovine (Sanguinate, Prolong Pharmaceuticals), has anti-vaso–constrictive properties which aid in the transfer of oxygen to oxygen-deprived cells and tissues. It is currently the only product in development designed to target comorbidities associated with sickle cell disease, such as vaso-occlusive crisis, acute chest syndrome, leg ulcers and pediatric and adult stroke.
PEGylated carboxyhemoglobin bovine is currently being evaluated in a phase 2 study for the reduction or prevention of delayed cerebral ischemia following subarachnoid hemorrhage. Phase 2 trials of PEGylated carboxyhemoglobin bovine also are planned to evaluate vaso-occlusive crisis and leg ulcers secondary to sickle cell disease and for preventing delayed graft function following kidney transplantation.
The product is also being evaluated internationally for the treatment of beta-thalassemia.
"Our Orphan Drug Designation has been issued based in part on Sanguinate's unprecedented ability to un-sickle sickle cells," Glenn Kazo, MS, president of Prolong Pharmaceuticals, said in a press release. "We now have multiple clinical studies underway where we hope to establish Sanguinate’s safety and efficacy in [sickle cell disease] and other diseases that are caused by the effects of oxygen deprivation."
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.