FDA Grants Orphan Drug Status To Two New Therapies In a Row, And These Two Groups Of Patients Are Expected To Benefit!
Recently, the US FDA has granted orphan drug status to two new therapeutics in a row. One is a small molecule inhibitor, PCLX-001, for the treatment of acute myeloid leukaemia; the other is a T-cell therapy, ET140203, for the treatment of hepatoblastoma. Both therapies are currently in relevant trials.