FDA grants Poolbeg’s POLB 001 Orphan Drug Status for CRS

Published 27/05/2025, 07:04
FDA grants Poolbeg’s POLB 001 Orphan Drug Status for CRS

LONDON - Poolbeg Pharma PLC (AIM:POLB), a clinical-stage biopharmaceutical company, announced on Tuesday that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to its drug candidate POLB 001. The designation is for the prevention of Cytokine Release Syndrome (CRS), a potentially fatal side effect associated with cancer immunotherapy treatments.

The FDA’s ODD program provides incentives to encourage the development of drugs for rare diseases, which affect fewer than 200,000 individuals in the U.S. Benefits of ODD include seven years of market exclusivity upon approval, potential fee waivers, and tax credits for clinical research.

POLB 001, an oral p38 MAP kinase inhibitor, is poised to enter Phase 2a clinical trials in the second half of 2025, with interim results expected in the first half of 2026 and complete data by the second half of the same year. The drug aims to address the unmet need for a preventative therapy for CRS, which currently affects over 70% of patients undergoing certain cancer immunotherapies.

The company has indicated that major pharmaceutical firms have shown interest in providing the bispecific antibody required for the Phase 2a trial at no cost, which underscores the industry’s recognition of POLB 001’s potential.

Jeremy Skillington, CEO of Poolbeg Pharma, expressed that the FDA’s decision enhances the drug’s commercial prospects and could expedite its path to market. Skillington also emphasized the potential benefits for patients, healthcare systems, and broader access to cancer immunotherapies.

Professor Brendan Buckley, a Non-Executive Director and member of the Scientific Advisory Board, also highlighted the importance of developing treatments for CRS, given the severe impact on patients.

Poolbeg Pharma is set to present further details about POLB 001 during a live presentation scheduled for Wednesday, May 28, 2025.

This report is based on a press release statement and contains no promotional content. It aims to provide investors with the essential information regarding Poolbeg Pharma’s recent FDA Orphan Drug Designation for POLB 001.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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