The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for Prolong Pharmaceuticals' flagship product Sanguinate for the treatment of sickle cell disease (SCD).
The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides the sponsor certain benefits and incentives, including a period of marketing exclusivity upon regulatory approval, potential tax credits for certain activities, eligibility for orphan drug grants, and the waiver of certain administrative fees.
"Our Orphan Drug Designation has been issued based in part on Sanguinate's unprecedented ability to un-sickle sickle cells," stated Glenn Kazo, president of Prolong Pharmaceuticals. "We now have multiple clinical studies underway where we hope to establish Sanguinate's safety and efficacy in SCD and other diseases that are caused by the effects of oxygen deprivation."
Dr Abraham Abuchowski, Prolong's CEO and chief science officer, said the designation underscored the partnership effort between the scientific research community and the regulatory authorities to ensure that those suffering from the devastating effects of SCD are not forgotten. "The Orphan Drug Act was designed to bring resources to bear on conditions and disease states which might not otherwise attract the efforts of private sector research," said Dr. Abuchowski, who in 1990 was responsible for the approval of the milestone drug Adagen for the treatment of Severe Combined Immunodeficiency Disease (SCID), commonly referred to as the "bubble boy" disease. Adagen was approved under the auspices of the Orphan Drug Act in 1990 based on trials with less than ten of the disease's worldwide patient population.
On March 16, 2015, Congressman Leonard Lance made a formal statement on the floor of the House of Representatives, applauding the orphan drug designation of Sanguinate for SCD while recognizing Dr. Abuchowski for "his contributions to the rare disease community and his important role in bringing innovative orphan therapies to market." In February of this year, the industry organization BioNJ awarded Dr. Abuchowski its annual Sol J. Barer Award for his track record of innovation in drug development and for his championing of rare disease research. A video on the award and Dr. Abuchowski's career may be viewed here: BioNj Honors Dr. Abuchowski
Sanguinate is an investigational biopharmaceutical product that through its anti-vaso-constrictive properties facilitates the transfer of oxygen to oxygen-deprived cells and tissues, and is the only biological product currently in clinical development for the multiple comorbidities of sickle cell disease (SCD). Many of the comorbidities of SCD are caused by a spiraling cycle of sickling, hemolysis and blood vessel inflammation. These comorbidities include vaso-occlusive crisis, acute chest syndrome, leg ulcers and pediatric and adult stroke. By correcting oxygen levels and down-regulating inflammation, Sanguinate has the promise of effectively treating many of the debilitating, acute comorbidities associated with SCD.
Phase I studies in healthy volunteers and stable SCD patients have been completed. Sanguinate is now in a Phase II study for the reduction or prevention of delayed cerebral ischemia following subarachnoid hemorrhage. Phase II trials are also planned for vaso-occlusive crisis and leg ulcers secondary to SCD as well as for preventing delayed graft function following kidney transplantation. The product is being evaluated for the treatment of beta-thalassemia in international trials.
Headquartered in South Plainfield, New Jersey, Prolong Pharmaceuticals, LLC is developing products to treat several diseases and their debilitating comorbidities associated with reduced quality of life, increased medical cost and significant mortality.