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Pluristem Therapeutics Announces FDA Orphan Drug Designation for PLX cell therapy
Sep. 25, 2018 08:50AM PST
Biotech InvestingPluristem Therapeutics (Nasdaq:PSTI) (TASE:PSTI), a leading regenerative medicine company developing novel placenta-based cell therapy products, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery following hematopoietic cell transplantation (HCT). As quoted in the press …
Pluristem Therapeutics (Nasdaq:PSTI) (TASE:PSTI), a leading regenerative medicine company developing novel placenta-based cell therapy products, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery following hematopoietic cell transplantation (HCT).
As quoted in the press release:
“We believe the FDA’s decision to grant Orphan Drug Designation to PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery reflects the potential of this regenerative therapy to improve outcomes in this underserved patient population,” said Yaky Yanay, Co-CEO and President of Pluristem. “We look forward to data from our ongoing Phase 1 clinical trial as we work to efficiently advance this novel therapy through clinical development for this and other hematopoietic indications.”
Pluristem’s product PLX-R18 is currently being evaluated for the treatment of insufficient hematopoietic recovery following bone marrow transplantation in an ongoing Phase 1 clinical trial in the U.S. and Israel. The trial is designed to evaluate the safety of intramuscular (IM) injections of PLX-R18 cells in 24 subjects with incomplete hematopoietic recovery persisting for at least 4 months after HCT, with a 12-month follow-up period. The primary endpoint is safety. Exploratory endpoints include changes in platelet and hemoglobin levels, changes in transfusion frequency, a shift from transfusion dependence to transfusion independence, changes in quality of life, and changes in the serum immunological parameters.
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders. Orphan drug designation provides to the Company certain benefits, including market exclusivity upon regulatory approval, if received, exemption of FDA application fees and tax credits for qualified clinical trials.