Cellphire, the global leader in long-term stabilization and storage of platelets, announced today that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its platelet-based freeze-dried hemostatic, Thrombosomes®, for treatment of acute radiation syndrome (ARS).
“The orphan drug designation is an important regulatory milestone as we further our development of Thrombosomes for the treatment of ARS,” said G. Michael Fitzpatrick, Ph.D., President of Cellphire. “Cellphire is committed to taking Thrombosomes, a next generation blood product, through the approval process in order to enhance our nation’s response capability.”
Orphan drug designation is granted by the FDA to drugs and biologics which are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.
Thrombosomes’ development has been supported by the Biomedical Advanced Research and Development Authority (BARDA), part of the office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services. BARDA invests in the innovation, advanced research and development, acquisition, and manufacturing of medical countermeasures– vaccines, drugs, therapeutics, diagnostic tools, and non-pharmaceutical products needed to combat health security threats including a radiation or nuclear incident.
Such an emergency would place an immediate burden on the blood supply. Blood products will be needed to treat the hemorrhage and cytopenias (low platelet counts) associated with ARS. Mitigating this sudden demand cannot be done by stockpiling traditional blood products due to their limited shelf life. Next generation blood products, like Thrombosomes, are needed to support the nation’s response capability.
Thrombosomes are lyophilized (freeze-dried) activated platelets. Unlike standard liquid platelets that can only be stored for 5-7 days after collection, Thrombosomes can be stored for up to 3 years at ambient temperatures. In March, the FDA approved a phase 2 clinical trial of Thrombosomes in bleeding thrombocytopenic patients. Cellphire expects to begin recruiting patients in the fourth quarter of 2020.
Cellphire, Inc. is a biotechnology company developing next-generation therapeutics. The company is applying its proprietary cell stabilization technology to all cell types, including platelets, to develop lifesaving products. Its lead product is Thrombosomes®, a freeze-dried hemostatic derived from human platelets. Cellphire’s technology has potential applications across a wide range of medical applications from trauma to wound care, imaging, targeted drug delivery, and regenerative medicine. For more information, visit www.cellphire.com.
The Thrombosomes project has been funded in whole or in part with federal funds from the Biomedical Advanced Research and Development Authority (BARDA), Office of the Assistant Secretary for Preparedness and Response, Office of the Secretary, Department of Health and Human Services, under Contract No. HHSO100201300021.
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