Novel CRS Prevention Therapy Granted FDA Orphan Drug Designation
POLB 001 has been granted FDA orphan drug designation as a preventative therapy for cytokine release syndrome caused by bispecific antibody therapy.
US FDA
- The FDA has granted orphan drug designation (ODD) to POLB 001.
- POLB 001 is an oral preventative therapy for bispecific T-cell engager (BiTE) therapy-induced cytokine release syndrome (CRS).
- The first patient is expected to be dosed with POLB 001 in a phase 2a study this year.
POLB 001, a preventative therapy for CRS caused by BiTE therapy, has been granted ODD by the FDA.1
ODD is intended to support the development of agents for rare disorders that affect less than 200,000 patients in the US. With this designation, Poolbeg Pharma, the sponsor, is eligible for a potential 7 years of market exclusivity following regulatory approval, potential exemption from Prescription Drug User Fee Act application fees, and tax credits for qualifying clinical trials.
According to real-world data presented at the 2024 American Society of Hematology (ASH) Annual Meeting, CRS is reported in nearly 90% of cases of patients treated with bispecific antibodies, signaling an unmet need in the treatment landscape.2
“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. 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,” said Jeremy Skillington, PhD, chief executive officer of Poolbeg Pharma, in a press release.1
POLB 001 is a phase 2-ready oral p38 MAP kinase inhibitor. Poolbeg expects to dose the first patient in a phase 2a study in the second half of 2025, with an interim analysis expected in the first half of 2026 and topline data from phase 2a in the second half of 2026.
"Orphan drug designation from the FDA underscores the urgency and importance of developing innovative therapies for this critical unmet medical need. We look forward to progressing POLB 001 in our upcoming phase 2a clinical trial and working closely with prospective partners and regulatory agencies to bring this potential therapy to patients as quickly as possible,” said Brendan Buckley, MD, director and scientific advisory board member at Poolbeg, in the press release.
