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America Stem Cell, ASC-101 Orphan Drug Designation

The FDA has granted Orphan Drug Designation to America Stem (NYSE:ASC) Cell’s ASC-101 to improve homing to bone marrow (treatment of myeloablation) in patients receiving hematopoietic stem cell transplantation.
  • ASC is commencing a Phase I/IIa clinical trial for the treatment of advanced hematological cancers at the University of Texas M.D. Anderson Cancer Center in Houston.

ASC-101 and ASC-201 are designed to improve the engraftment of stem cells to target organs and increase their therapeutic potential. Hematopoietic stem cells derived from cord blood, peripheral blood, and bone marrow is used to treat 70+ diseases.

The Bottom Line: America Stem Cell (ASC) is a privately held company dedicated to the development and commercialization of enabling technologies to enhance and expand the therapeutic potential of stem cell transplants. ASC has established a number of strategic partnerships with medical research institutions including the University of Texas M.D. Anderson Cancer Center, the Oklahoma Medical Research Foundation, and VidaCord Technologia Biomedica. Orphan drug status confirms the importance of this new novel treatment approach to improve outcomes for patients with serious, life-threatening cancers and other disorders for which hematopoietic stem cell transplant is prescribed. The  designation will confer financial benefits and incentives, such as potential grant funding to defray the cost of clinical testing, tax credits for the costs of clinical research, 7 year period of exclusive marketing after approval and Waiver of Prescription Drug User Fee Act (PDUFA) filing fees.