Prescient Therapeutics Ltd (ASX:PTX) is trading higher on being granted additional Orphan Drug Designation for PTX-100 for the treatment of T-cell lymphomas (TCL), including cutaneous TCL (CTCL).
The designation from the Office of Orphan Products Development at the US Food and Drug Administration (FDA), follows ODD for peripheral TCL (PTCL) in 2022, when Prescient applied for ODD for CTCL.
The latest designation broadens the clinical stage oncology company’s range to all TCLs. This means the ODD designation now covers all TCLs and their subtypes.
It has drawn a positive response from investors with shares as much as 28.87% in early trading on the ASX to A$0.125.
The FDA’s ODD program provides orphan status to drugs which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US. It is designed to provide benefits to incentivise drug development in less common diseases.
What are TCLs?
TCLs describe a group of lymphomas that develop when a group of 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.
PTX is currently focused on relapsed and refractory TCLs for the current expansion cohort of the PTX-100 Phase 1b study.
The study is under the leadership of globally renowned lymphoma expert, Professor H Miles Prince, AM.
An update of the trial is due soon.