Prescient Therapeutics Ltd (ASX:PTX)’s PTX-100 for the treatment of peripheral T-cell lymphomas (PTCL) has been granted orphan drug status by the US Food and Drug Administration (FDA).
The orphan status program is designed to provide incentives for the development of drugs to treat rare diseases that affect fewer than 200,000 people in the US.
The designation brings considerable benefits including guaranteed market exclusivity for seven years and a waiver of Prescription Drug User Fee Act fees for orphan drugs, valued at over US$3.1 million in 2022.
Significant milestone
“The granting of Orphan Drug Designation by the FDA is significant for Prescient’s development of PTX-100,” Prescient managing director and CEO Steven Yatomi-Clarke said.
“Orphan drugs often enjoy shorter and cheaper development pathways. Additionally, the company now has the certainty of seven years of market exclusivity in the event of regulatory approval of PTX-100 for PTCL.
“PTCL is a disease of unmet need, where safer and more effective therapies will be welcomed by patients and clinicians. As our expansion cohort unfolds, ultimately it will be supporting clinical data and Orphan Drug Designation that will combine to help bring PTX-100 to patients with this challenging disease.”
Encouraging efficacy
PTX-100 has shown encouraging efficacy in PTCL in the dose escalation of a Phase 1b basket study, which has been expanded to include 12 patients with relapsed and refractory T cell lymphomas (TCL), including PTCL.
The study is being led by globally-renowned lymphoma expert Professor H. Miles Prince, AM.
Prescient notes that survival from PTCL following relapse is poor and treatment has not significantly improved in the last 20 years.
While the disease is not a common malignancy, its nature and the paucity of effective treatment options for refractory patients creates a potentially shorter regulatory path for PTX-100, and the designation is the fastest route to bring the drug to market.