The United States Food and Drug Administration has awarded Orphan Drug Designation to an experimental PLX cell therapy (called PLX-R18) as a treatment for graft failure and incomplete haematopoietic recovery after haematopoietic cell transplantation. For more detailed information you can view the source press release here, at Globe Newswire.
About Haematopoietic Cell Transplantation (HCT)
HTC is an infusion of certain cells that are used to establish marrow and immune function in patients and may be used as a therapy for a range of conditions, including some cancers and genetic disorders. The transplanted cells can come from the patient themselves (an autologous transplantation), or a matched donor (an allogeneic transplantation).
PLX-R18 is a cell therapy that is being investigated by Pluristem Therapeutics as a potential treatment for patients who experience graft failure or incomplete haematopoietic recovery after HCT. The FDA has recently granted the experimental therapy Orphan Drug Designation for this use.
About Orphan Drug Designation
Orphan Drug status is awarded by the FDA to certain drugs that are being developed to treat rare disorders. The designation is designed to encourage and speed up the drug development process, through offering incentives to eligible drugs such as a longer period of marketing exclusivity, tax credits, and waived drug user fees. For more information about Orphan Drugs, click here.
Research into PLX-R18
An ongoing Phase 1 trial is investigating PLX-R18 in patients who have had insufficient haematopoietic recovery after bone marrow transplantation. The study is taking place in the US and Israel and involves 24 patients. The study is designed to assess the safety of the therapy, and, to a lesser extent, other factors such as how it affects platelet and haemoglobin levels, and patients’ quality of life. Mr Yanay, the President and Co-CEO of Pluristem, says, “we look forward to data from our ongoing Phase 1 clinical trial.”