Biopharmaceutical company Poolbeg Pharma said the US Food and Drug Administration has granted Orphan Drug Designation (ODD) to its POLB 001 product as an oral preventative therapy for Cytokine Release Syndrome (CRS).
Cancer immunotherapy-induced CRS is a severe side-effect that occurs in more than 70% of patients and which may lead to multi-organ failure and death. There are currently no approved preventative therapies for CRS.
Poolbeg focusses on the development of innovative medicines to address unmet medical needs and its programmes target large addressable markets including, cancer immunotherapy-induced Cytokine Release Syndrome (CRS) and metabolic conditions such as obesity.
The company said the FDA grants orphan status to support the development of medicines for rare disorders affecting less than 200,000 people in the US.
It provides Poolbeg with clinical development and commercialisation benefits including the potential for a seven-year period of US market exclusivity after regulatory approval of POLB 001, potential waiver exemption of Prescription Drug User Fee Act application fees and the potential for tax credits for qualifying clinical trials.
Poolbeg expects the first patient to be dosed in its proposed Phase 2a in the second half of this year, with interim analysis expected in the first half of 2026 and Phase 2a topline data in the second half of 2026.
Jeremy Skillington, the chief executive of Poolbeg Pharma, said that POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients’ lives.
“We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster,” he said.
“If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies,” the CEO added.
