Prescient Therapeutics announced that the Office of Orphan Products Development at the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTX-100 for the treatment of T-cell lymphomas (TCL), including cutaneous TCL (CTCL). Having separately received ODD for peripheral TCL (PTCL) in 2022, Prescient applied for ODD for CTCL. The FDA has now granted a broader designation than Prescient requested, which encompasses all TCLs.

TCLs describes a group of lymphomas that develop when a group white blood cells called lymphocytes grow out of control. There are different groups of TCL, including PTCL and CTCL, each with several distinct subtypes. Collectively, TCLs represent an area of unmet or poorly met patient need, especially in patients with relapsed or refractory disease.

This ODD designation now covers all TCLs and their subtypes. The benefits of an Orphan Drug Designation are considerable and include guaranteed market exclusivity of seven years from granting of regulatory approval; and a waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, which has a value of over USD 3.1 million in 2022. Orphan Drug Designation will allow Prescient to benefit from incentives that can assist the development of PTX-100, a first-in-class prenylation inhibitor that disrupts oncogenic Ras pathways in cancer cells.