Poolbeg Pharma PLC (AIM: POLB) has received a significant regulatory endorsement from US authorities for one of its experimental treatments, which aims to address a serious side effect of advanced cancer therapies.
The London-listed biotech announced on Tuesday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to POLB 001, a potential treatment intended to shield patients from cytokine release syndrome (CRS).
Cytokine release syndrome (CRS), a frequent and potentially life-threatening side effect of some cancer immunotherapies, causes an intense immune response that can result in high fever, organ failure, and even death.
In the US, Orphan Drug Designation is granted to treatments for conditions affecting fewer than 200,000 people and comes with a range of incentives. These include tax credits, waived regulatory fees, and seven years of market exclusivity if the treatment receives approval.
Poolbeg Pharma CEO Jeremy Skillington described POLB 001 as a potentially game-changing, orally administered preventative therapy for immunotherapy-induced CRS. “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 its commercial appeal to potential partners and accelerate its path to market,” he said.
“If approved, POLB 001 could improve patients’ quality of life, ease the burden on healthcare systems, and broaden access to cancer immunotherapies.”
POLB 001 works by targeting the p38 MAP kinase protein, a key driver of the inflammatory process linked to CRS. The drug is being developed as a tablet, and Poolbeg plans to initiate a mid-stage clinical trial in the second half of 2025, with early results expected in the first half of 2026 and final data by year-end.
The company also reported strong interest from major pharmaceutical firms willing to supply the specific antibodies used in cancer treatments that trigger CRS. Providing these antibodies free of charge would help lower development costs and reflect industry confidence in Poolbeg’s approach.
Currently, no approved preventative therapies exist for CRS. Poolbeg believes that POLB 001 could make advanced cancer treatments safer and more accessible, potentially enabling wider use in outpatient settings.
