Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its 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.
Prolong Pharmaceuticals LLC