PLX cells regulatory update

Pluristem said FDA requested additional information and data to determine if the company's allogeneic, placental-derived expanded (PLX) cells qualify for Orphan Drug designation to treat aplastic anemia. According to the company, the agency acknowledged that aplastic anemia qualifies as an Orphan indication but requested evidence that the product is an appropriate therapy. Pluristem told BioCentury that it has already accumulated the undisclosed information and plans to submit the data "in a timely manner." The PLX cells are in preclinical testing for hematology indications, including aplastic anemia.

According to FDA, the agency has granted Orphan Drug designation to 1 compound for aplastic anemia for which the designation has not been withdrawn - etiocholanedione from SuperGen Inc., which Astex Therapeutics Ltd. acquired and was subsequently renamed Astex Pharmaceuticals Inc. FDA granted Orphan Drug designation to 2 other compounds, but withdrew the designation. ...