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Highlights

  • Poolbeg Pharma’s POLB 001 receives Orphan Drug Designation from the US FDA.

  • The drug aims to prevent cytokine release syndrome (CRS), a dangerous side-effect of cancer immunotherapies.

  • Designation provides tax incentives, fee waivers, and 7 years of US market exclusivity.

  • Mid-stage clinical trial for POLB 001 to begin in H2 2025; headline results expected by end of 2026.

  • Poolbeg in talks with major pharma companies to supply key antibodies for trial at no cost.

Poolbeg Pharma PLC (LSE:POLB) has achieved a major regulatory milestone in the United States, announcing that the Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its lead candidate POLB 001, an oral therapy developed to prevent cytokine release syndrome (CRS).

CRS is a serious and potentially fatal immune reaction often triggered by advanced cancer immunotherapies. It can result in severe inflammation, high fever, multi-organ failure, and in some cases, death. Despite the growing use of immune-based cancer treatments, no preventive therapies are currently approved to address CRS.

Jeremy Skillington, CEO of Poolbeg, described POLB 001 as a "potential breakthrough" in protecting cancer patients undergoing immunotherapy, noting that the FDA’s orphan designation could accelerate development and enhance commercial appeal to prospective partners.

“We believe POLB 001 has the potential to improve quality of life, reduce healthcare burdens, and make immunotherapies more accessible,” Skillington said.

POLB 001 works by blocking the p38 MAP kinase protein, a key player in the inflammatory response that causes CRS. The drug is in tablet form and is designed to be taken proactively, which could make immunotherapies safer for outpatient use.

Regulatory & Commercial Benefits

Orphan Drug Designation, reserved for treatments targeting diseases affecting fewer than 200,000 people in the US, offers a suite of regulatory and financial incentives:

  • Seven years of market exclusivity upon approval

  • Tax credits on clinical development costs

  • Waived FDA application fees

Poolbeg is preparing to launch a Phase II trial in the second half of 2025, with early clinical readouts in H1 2026 and final headline data expected by year-end. Notably, the company is gaining interest from larger pharmaceutical firms offering to supply critical antibody therapies used to trigger CRS in trials, a move that could lower costs and validate Poolbeg’s approach.