Placenta based cell therapy product PLX-R18 has been granted orphan drug designation by the FDA. The drug’s developers, Pluristem, have a potentially expedited pathway to approval, as well as extended exclusivity and tax credits as a result of the decision.
PLX-R18 secretes proteins which trigger the regeneration of hematopooietic cells in bone marrow, and hence the production of red blood cells. This has been designed to treat patients with certain cancers or cancer treatments, immune-mediated bone marrow failure, or acute radiation syndrome (ARS)- the latter of which is the subject of the orphan designation.
In vivo testing of PLX-R18 to date has demonstrated improvement in survival rates and improved cell recovery across all three major blood lineages. Pluristem is preparing for a human trial in ARS, and is currently enrolling patients in a Phase I trial in incomplete bone marrow recovery following hematopoietic cell transplantation (HCT). U.S. Department of Defense’s (DOD) Armed Forces Radiobiology Research Institute (AFRRI) and Fukushima Medical University in Japan are also conducting investigations into the therapeutic cells.
‘Pluristem has a vast and dynamic program developing our PLX-R18 therapy as a treatment for ARS, which can potentially save many lives. Receiving Orphan Drug Designation brings us one step closer to providing a next-generation medical countermeasure against ARS, which is especially important given today’s volatile political climate.’ commented CEO Zami Aberman.
Source: FDA Grants Pluristem Orphan Drug Designation for Its PLX-R18 Cell Therapy as Treatment for Acute Radiation Syndrome Press Release